Reported trial results for Haemophilia

132 trials have reported results.

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

• NCT04244981 · results posted 24 June 2026

  According to the results reported on ClinicalTrials.gov, this trial (NCT04244981) enrolled 476 people who were undergoing surgery and needed help managing bleeding. Participants were split into two groups: 240 received a treatment called PCC (a concentrated clotting factor product) and 236 received FFP (fresh frozen plasma, a blood product that also contains clotting factors). The trial was looking at bleeding and clotting-related outcomes after surgery, with the main measure being how much fluid drained through a chest tube in the 24 hours following surgery. The reported data shows that, for the primary measure — cumulative chest tube drainage — the PCC group had an average of 482 ml, while the FFP group had an average of 477 ml. For one of the secondary measures, the number of participants in whom no additional bleeding interventions were needed between one and 24 hours after treatment was 196 out of 240 in the PCC group, and 169 out of 236 in the FFP group. The reported data for red blood cell transfusions shows a value of zero units in both groups. Two other pre-specified outcome measures — changes in blood test results (such as clotting times and blood counts) and length of hospital or ICU stay — were listed in the trial registration but no numerical results were reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT06543368 · results posted 27 February 2026

  According to the results reported on ClinicalTrials.gov, this trial enrolled 14 participants who each received a PRP (platelet-rich plasma) injection into a joint affected by osteoarthritis. PRP is a treatment made from a person's own blood that is processed and injected back into the body. The trial looked at joints in different parts of the body — 5 knees, 9 ankles, and 3 elbows were assessed among the 14 participants (noting that some participants may have had more than one joint treated). All 14 participants completed the trial. The study measured how satisfied participants were with their experience six months after the injection, as well as scores related to pain, stiffness, and daily function depending on which joint was treated. The reported data shows that participants rated their overall satisfaction with the injection at an average score of 70 out of 100, where 0 meant "not satisfied at all" and 100 meant "completely satisfied." For the knee-specific questionnaire (called WOMAC, which measures pain, stiffness, and physical function on a scale where higher numbers mean worse symptoms), the reported total score was 31 out of a possible 96. The reported sub-scores were 7 for pain (out of 20), 4 for stiffness (out of 8), and 21 for physical function (out of 68). For participants with elbow or upper-limb involvement, a questionnaire called the QuickDASH — which measures arm and hand disability on a 0–100 scale where higher means more difficulty — reported an average score of 22.73. For ankle participants, a foot and ankle function score (FAOS) was reported at 94.12 out of 100, where 100 represents no functional limitations. It is important to note that this was a small study with only 14 participants and no comparison group, so the numbers above simply describe what was recorded in those individuals. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05036278 · results posted 12 November 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT05036278) enrolled 21 participants, with 19 completing the study and 2 not completing it. The trial was looking at a treatment called damoctocog alfa-pegol (brand name Jivi), a longer-acting clotting factor product, given as a preventive (prophylaxis) regimen to people with haemophilia A. Participants had previously been on a standard shorter-acting product, and the study used a risk score based on each person's individual health information to assign them to a Jivi dosing schedule. The main thing being measured was how many participants had a "favourable outcome" on their assigned regimen. The reported data shows that 12 out of the 21 participants who started the study were recorded as having a favourable outcome on their assigned Jivi regimen. For the secondary measures, the reported average number of bleeds per year was approximately 4.2 overall, 2.8 for joint bleeds, and 1.8 for spontaneous (unprompted) bleeds. Compared to before the study, the reported data shows an average change of roughly minus 8 bleeds per year, suggesting participants experienced fewer bleeds on average than they had on their previous treatment — though this is simply what the numbers show, not a conclusion about why. The reported data also shows that the average number of infusions per month decreased by about 6.9 compared to the previous treatment. Thirteen out of 19 completing participants recorded either zero or no more than one spontaneous bleed during the study. On a quality-of-life questionnaire (where higher scores mean greater difficulty), the reported average score was 31.5 out of 100, with an average change from the start of the study of minus 4.7 points. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03587116 · results posted 6 November 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT03587116) enrolled 212 people in total — 111 with haemophilia B and 101 with haemophilia A — all of whom were receiving standard clotting factor replacement therapy. The trial was an observational study, meaning it did not test a new treatment but instead tracked and recorded how often participants experienced bleeding episodes while on their existing therapy. The main thing being measured was each person's **annualised bleeding rate (ABR)** — that is, the average number of bleeding episodes per year that either were treated or went untreated. The reported data shows the following bleeding rates for the **haemophilia B** group during the prospective (forward-looking) data collection period: approximately **3.61 treated bleeds per year** and **4.46 total bleeds per year** (efficacy analysis set); **3.71 treated** and **4.56 total** bleeds per year (per-protocol set); and **4.21 treated** and **4.78 total** bleeds per year (protocol amendment 5 set). When data collected retrospectively (looking back in time) was included for the amendment 5 group, the reported figures were higher — **5.42 treated** and **6.79 total** bleeds per year; and when retrospective and prospective data were combined, **4.65 treated** and **5.48 total** bleeds per year. For the **haemophilia A** group, the reported data shows **4.87 treated bleeds** and **6.10 total bleeds** per year (efficacy analysis set, prospective period). Bleeding rate figures for the haemophilia A group under the other analysis sets were not reported in the data provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04082429 · results posted 31 October 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04082429) tested a medicine called concizumab in people living with haemophilia A or haemophilia B — inherited conditions where blood does not clot properly. The trial enrolled participants across several groups. For haemophilia A, the main comparison involved 9 people who received no preventive (prophylaxis) treatment and 18 people who received daily concizumab injections as a preventive measure. For haemophilia B, 12 people received no preventive treatment and 24 received concizumab as prevention. Additional groups of participants who had previously been on concizumab in an earlier related study were also included. The trial's main focus was measuring how often treated bleeding episodes (both spontaneous and those caused by injury) occurred per year in each group — a figure known as an annualised bleeding rate (ABR), meaning the estimated average number of treated bleeds per person per year. The reported data shows the following primary (main) outcome numbers. For haemophilia A participants, those receiving no preventive treatment had a reported ABR of 19.6 treated bleeds per year, while those receiving concizumab as prevention had a reported ABR of 2.9 treated bleeds per year. For haemophilia B participants, those receiving no preventive treatment had a reported ABR of 14.9 treated bleeds per year, while those receiving concizumab as prevention had a reported ABR of 1.6 treated bleeds per year. The reported data also shows secondary (additional) outcomes measuring spontaneous bleeds only: for haemophilia A, the no-treatment group reported 19.3 per year versus 1.0 per year in the concizumab group; for haemophilia B, 10.8 per year versus 1.0 per year respectively. A separate secondary analysis looking at participants who had already been on stable concizumab prevention from a prior study reported ABRs of 2.2 (prior study) and 1.7 (this study) for haemophilia A, and 2.1 (prior study) and 1.3 (this study) for haemophilia B. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03061201 · results posted 30 September 2025

  According to the results reported on ClinicalTrials.gov, this trial tested a gene therapy called PF-07055480 in 11 people across four groups, each receiving a different dose. The groups are referred to as Cohorts 1 through 4, with each cohort receiving a progressively higher dose. The trial was measuring two main things: how many participants experienced any medical events (called adverse events) or serious medical events (serious adverse events) after receiving the treatment, and how much of a blood-clotting protein called Factor VIII (FVIII) could be detected in participants' blood over time — up to five years. Factor VIII is a protein that people with haemophilia A have too little of. The results for Cohort 1 (the lowest dose group) were not reported for the FVIII activity measurements, and data was not reported for all time points across all groups. The reported data shows that across all four groups, every participant who started the study experienced at least one adverse event. For serious adverse events specifically, no participants in Cohorts 1 or 3 had one recorded, two participants in Cohort 2 did, and one participant in Cohort 4 did. For FVIII activity levels — measured as a percentage of what would be considered normal — the reported data shows varying results across the three cohorts where data was available. In Cohort 2, the reported level went from 0.90% at Year 1, rose to 19.30% at Year 2, then gradually fell to 0.90% by Years 3, 4, and 5. In Cohort 3, levels went from 11.90% at Year 1 to between roughly 3% and 8% across the following years. In Cohort 4 (the highest dose), levels started at 42.60% at Year 1 and remained roughly in the range of 23% to 27% through to Year 5. It is worth noting that these groups were very small — between 2 and 5 people each — so the numbers reflect only a handful of individuals. No data was reported for Cohort 1 across the FVIII activity measurements, and not all participants completed the study. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03370172 · results posted 11 September 2025

  According to the results reported on ClinicalTrials.gov, this trial tested an experimental gene therapy called BAX 888 in people with haemophilia A — a condition where the blood does not clot properly due to low levels of a protein called Factor VIII (FVIII). The trial was very small, with just four participants in total: two people received a lower dose of BAX 888, and two received a higher dose. The main thing the trial was set up to measure was how many participants experienced unwanted medical events (called adverse events) that were linked to BAX 888. Secondary measurements looked at changes in FVIII levels in the blood, how often participants had bleeding episodes, and whether they needed to use less of their usual FVIII replacement treatment. The reported data shows that all four participants — both in the lower-dose group and the higher-dose group — experienced at least one adverse event considered related to BAX 888. Regarding FVIII activity levels in the blood, the lower-dose group showed an average increase of 11.40 units per decilitre from their starting level, while the higher-dose group showed an average increase of 248.30 units per decilitre. For clinically significant changes in FVIII protein levels, the reported data shows this was observed in 2 participants in the lower-dose group and 0 in the higher-dose group. The reported annualised bleed rate (an estimate of how many bleeds per year) was 1.0 for the lower-dose group and 0.5 for the higher-dose group. Notably, the reported data shows that 0% of participants in either group used less of their usual FVIII replacement treatment during the study period. No participants in either group developed antibodies that would block FVIII from working. It is worth noting that one participant in the higher-dose group did not complete the study, and with only four participants overall, this trial was a very early-stage study involving a very small number of people. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04370054 · results posted 17 August 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04370054) enrolled 77 participants, all of whom received a single infusion of an investigational treatment called PF-07055480. Seventy-five participants completed the treatment phase. The trial was measuring bleeding rates, levels of a clotting protein called Factor VIII (FVIII) in the blood, and how much additional clotting treatment participants needed — both before receiving PF-07055480 (while they were on their usual preventive clotting factor treatment) and after receiving it. The reported data shows that, before receiving PF-07055480, participants had a reported average of 4.73 total bleeds per year and 4.08 bleeds per year that required treatment with a clotting factor. After receiving PF-07055480, those figures were reported as 1.24 total bleeds per year and 0.07 treated bleeds per year. The reported data also shows that before the infusion, participants were receiving an average of around 124 separate clotting factor infusions per year; after receiving PF-07055480, that figure dropped to around 0.21 infusions per year. At 15 months after the infusion, 84% of participants were reported to have FVIII activity levels above 5% (a measurement of how much clotting protein was present in the blood). The average FVIII activity level measured between week 12 and 15 months after infusion was reported as 96.84% of normal using one measurement method, with a second measurement method reporting 134.95% of normal. Annual consumption of clotting factor (measured in units per kilogram of body weight) was reported as 4,082.7 before the infusion and 6.6 after. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05437211 · results posted 5 June 2025

  According to the results reported on ClinicalTrials.gov, this trial looked at whether using a Virtual Reality (VR) headset during home infusions of clotting factor (Factor VIII or Factor IX, used to treat haemophilia) could change levels of anxiety and pain in children and their carers. A total of 24 people started the trial — 14 in a group that could control the VR experience themselves ("With Autonomy") and 10 in a group that could not ("Without Autonomy"). Two people, one from each group, did not complete the study, leaving 22 who finished. The trial measured anxiety and pain using simple 0–10 rating scales (where 0 means no anxiety or pain and 10 means extreme anxiety or pain), checked before and after each infusion over roughly four weeks. The reported data shows that average anxiety scores before infusions were low across both groups. For the "With Autonomy" group, two anxiety score values were reported — 0.2 and 1.5 — at different time points, while the "Without Autonomy" group had a reported score of 0.0 (the data as submitted does not fully clarify which time points each figure corresponds to). When looking at all participants together, 5 out of 22 were reported to have had their anxiety score drop by 2 or more points out of 10 across the study period. For pain after infusions, the "With Autonomy" group reported an average score of 1.4 out of 10, and the "Without Autonomy" group reported 0.6 out of 10. Across all participants combined, 4 out of 22 were reported to have had their pain score drop by 2 or more points out of 10. The reported data shows the trial was small in size, and some details in the submitted results — such as which specific time points certain scores belong to — were not fully clear from the data as reported. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05707351 · results posted 1 May 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT05707351) involved 37 participants who received a treatment called Adynovate, which is a clotting factor product used in people with haemophilia A. The trial was measuring how often bleeding episodes occurred while participants were on a regular (preventive) dosing schedule, as well as how much of the treatment was being used. Of the 37 people who started the study, 34 completed it and 3 did not. The reported data shows that, on average, participants experienced approximately 4.1 bleeding episodes per year in total. When broken down by location, the reported rate was 2.7 bleeds per year in joints and 1.4 bleeds per year in non-joint areas. Looking at what triggered the bleeds, the reported data shows a rate of 3.8 bleeds per year that were spontaneous or had no known cause, and 0.3 bleeds per year linked to an injury. These are averages across the group and individual experiences varied. The reported data also shows that, on average, participants used Adynovate roughly 2 infusions per week (about 8.7 per month), with a weight-adjusted dose of approximately 89.6 international units per kilogram of body weight per week. These figures describe how the treatment was being administered during the study period. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04083781 · results posted 27 April 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04083781) enrolled 133 people in total across four groups. Participants either had haemophilia and were not receiving any preventive (prophylaxis) treatment, or they were assigned to receive a medicine called concizumab as a daily preventive injection. The trial was primarily measuring how often treated bleeds — both spontaneous (happening without a clear cause) and injury-related — occurred per year, expressed as an "annualised bleeding rate" (ABR), which is simply the estimated average number of treated bleeds a person would have in one year. The reported data shows that in the group who received no preventive treatment (on-demand only), the ABR for treated spontaneous and traumatic bleeds was 9.8 events per year. In the group who switched to concizumab preventive treatment, the reported ABR was 0.0 events per year. For secondary measures, the reported rate of spontaneous bleeds alone was 8.4 per year in the no-prophylaxis group and 0.0 in the concizumab group. Reported joint bleed rates were 6.5 per year (no prophylaxis) versus 0.7 per year (concizumab group). The reported data also shows changes in self-reported physical wellbeing scores (measured on a standard quality-of-life questionnaire) over 24 weeks, with all four groups showing increases in scores for both bodily pain and physical functioning, ranging from 1.6 to 9.3 points depending on the group and measure. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03169972 · results posted 4 February 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled 135 people in total — 123 who had previously received treatment for their condition (called "Previously Treated Patients") and 12 who had not been treated before ("Previously Untreated Patients"). All 135 participants completed the study. The trial was measuring several things about a medicine called ADYNOVATE, including how many people stopped taking it, how often bleeding episodes occurred during a regular preventive (prophylaxis) dosing schedule, how long participants stayed on treatment, and what doses were used. The reported data shows that 10 of the 123 previously treated patients and 5 of the 12 previously untreated patients stopped taking the study medicine during the trial. For bleeding episodes that happened on a regular preventive schedule, previously treated patients had a reported rate of approximately 1.99 spontaneous (unprompted) bleeds per year, and 3.16 breakthrough bleeds per year; previously untreated patients had a reported breakthrough bleed rate of 2.91 per year. The spontaneous bleed rate for previously untreated patients was not reported in the submitted data. On the preventive schedule, previously treated patients were on the medicine for an average of 343 days, while previously untreated patients averaged 510.7 days. A smaller number of participants used the medicine "on demand" (only when needed rather than on a regular schedule), with previously treated patients averaging 13.3 days and previously untreated patients averaging 5.0 days on that approach. The reported average dose per injection on the preventive schedule was 39.4 units per kilogram of body weight for previously treated patients and 45.8 units per kilogram for previously untreated patients. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03003533 · results posted 30 December 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT03003533) tested an investigational gene therapy called SPK-8011 in a total of 25 adults with haemophilia A — a condition where the body does not make enough of a blood-clotting protein called Factor VIII (FVIII). Participants were split into four groups, each receiving a different dose of SPK-8011: 2 people in the lowest-dose group, 3 in the next, 9 in the third, and 11 in the highest-dose group. The trial was measuring things like how FVIII activity levels in the blood changed over time, how often bleeding occurred, how often participants needed extra FVIII infusions (top-up treatments), and what medical events (called adverse events) occurred after receiving the treatment. The reported data shows that, when looking at peak FVIII activity levels in the first 52 weeks — measured as a percentage of what is considered normal — the median figures were 11.5% for the lowest-dose group, 20.0%, 38.7%, and 55.1% for the higher-dose groups respectively. By week 52, the reported median steady-state FVIII levels were lower across all groups: 5.4%, 7.8%, 8.4%, and 4.1%. For spontaneous (unprompted) bleeding events, the annualised rate — meaning the estimated number per year — was reported as 0.3 for the second group, and 0.0 for the two higher-dose groups; the figure was not reported for the lowest-dose group. The annualised number of FVIII top-up infusions ranged from 0.3 to 7.4 across the groups. Regarding adverse events, all 25 participants experienced at least one treatment-emergent adverse event (a medical event occurring after receiving the study drug). Additionally, 0 participants in the lowest-dose group, 2 in the second, 7 in the third, and 10 in the highest-dose group received corticosteroid medicines — a type of anti-inflammatory drug — for a presumed immune response to the treatment. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04580407 · results posted 2 December 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04580407) looked at a treatment called TAK-672, which is a clotting factor product. The trial enrolled 5 participants in total, 4 of whom completed the study and 1 who did not. All participants had severe bleeding episodes, and the trial was measuring how well TAK-672 brought those bleeding episodes under control — checking things like whether bleeding stopped or reduced, and whether clotting factor levels in the blood reached certain targets. The reported data shows that, at 24 hours after treatment began, 100% of participants were recorded as having a "positive response" to TAK-672 — meaning their bleeding was rated by the investigator as either fully stopped or meaningfully reduced, with clotting factor levels meeting the pre-set thresholds. The reported data also shows that 100% of participants had their qualifying severe bleeding episode recorded as "successfully controlled" by the end of the initial treatment period. When responses were tracked at multiple time points across the study, the reported figures ranged from 80% at the earliest check to 100% at all later time points. On average, participants received approximately 1.8 infusions per day, with a total of around 2.6 infusions per participant needed to address the qualifying bleeding episode. The average total dose per participant was reported as 38,640 units. Because this trial involved only 5 people, the numbers reflect a very small group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04941898 · results posted 15 November 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 16 participants, all of whom received a PEGylated recombinant Factor VIII treatment given by intravenous infusion (a drip into a vein). Factor VIII is a clotting protein, and this type of treatment is used in people with haemophilia A, a condition where the blood does not clot properly. Fifteen of the 16 participants completed the study, and one did not complete it. The trial was measuring a number of things, including any unwanted medical events that occurred during the study, as well as how well bleeding was controlled during surgery. The reported data shows that 2 out of 16 participants experienced an adverse event — meaning an unwanted or unexpected medical occurrence that happened during the time they were in the trial. One participant experienced a serious adverse event, which is defined as one that was life-threatening, required hospitalisation, or caused significant disability. However, the reported data shows that zero participants experienced an adverse event considered to be directly related to the study drug itself, and zero participants experienced the specific serious reactions of inhibitor development (where the body produces antibodies against the treatment), shock, or anaphylaxis (a severe allergic reaction). For the secondary outcome looking at bleeding control during surgery, the reported data shows that across all surgeries performed, 93.3% were rated "Excellent" — meaning blood loss during the operation was no more than what would be expected in someone without haemophilia — and 6.7% were rated "Good," meaning blood loss was up to 50% more than expected. The reported data shows zero surgeries were rated "Fair" or "None." These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02137850 · results posted 31 October 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT02137850) involved children with haemophilia A — a condition where the blood does not clot properly due to low levels of a protein called Factor VIII. The trial looked at a treatment called N8-GP (also known as turoctocog alfa pegol), which is a modified form of that clotting protein. Participants were placed into one of three groups depending on their treatment stage: a pre-prophylaxis group (55 children started, 44 completed), a prophylaxis group — meaning regular preventive treatment — (69 children started the main phase, 55 completed it, and 49 then completed an extension phase), and a small immune tolerance induction group (8 children started, 4 completed). This last group was for children whose immune systems had started producing antibodies that block the clotting protein. The reported data shows that the primary thing being measured was how many children developed inhibitory antibodies — that is, antibodies that block the clotting protein from working. According to the results reported on ClinicalTrials.gov, 11 children in the pre-prophylaxis group and 10 children in the prophylaxis group developed these antibodies. Of those, 3 in the pre-prophylaxis group and 8 in the prophylaxis group had what are described as "high titre" inhibitors, meaning the antibody levels were above a particular threshold (5 Bethesda Units). For the prophylaxis group, the reported data shows an average of approximately 1.35 bleeding episodes per patient per year while on regular preventive treatment, and an average dose of 68.9 IU/kg was used for prophylaxis. The reported data also shows the number of adverse events (unexpected or unwanted medical occurrences during the trial) recorded across the groups. In the pre-prophylaxis group, 116 adverse events were recorded in total, including 24 classified as serious and 17 as events of special interest. In the prophylaxis group, 644 adverse events were recorded in total, including 56 serious and 47 of special interest. In the immune tolerance induction group, 32 adverse events were recorded in total, including 3 serious and none of special interest. The trial also tracked how well bleeding episodes were controlled using a four-point scale (excellent, good, moderate, or none) — the breakdown of individual bleeding episodes across these categories was recorded, but a single overall summary figure was not provided in the reported data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04541628 · results posted 4 September 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04541628) enrolled three participants, one in each of three groups (called Cohort 1, Cohort 2, and Cohort 3). Each person received at least one dose of the study drug, SIG-001. The trial was primarily measuring whether participants experienced any unexpected medical events (called "treatment emergent adverse events") after receiving the study drug. It also tracked things like changes in a clotting protein level in the blood (Factor VIII activity), the number of bleeding episodes over time, and how many doses of a replacement clotting treatment were needed. The reported data shows that all three participants — one in each cohort — experienced at least one treatment emergent adverse event. Regarding more serious adverse events, the data shows that one participant (in Cohort 3) had at least one serious treatment emergent adverse event, while the participants in Cohorts 1 and 2 did not. For the secondary outcomes, the reported data shows that one participant (Cohort 3) developed antibodies against the clotting protein being measured (detected by a laboratory test), while the participants in Cohorts 1 and 2 did not. Changes in clotting protein activity levels from the start of the study varied across the three participants. The number of bleeding events per year also varied across the groups and across different time periods within the study. The total number of doses of replacement clotting therapy used were reported as 401, 327, and 1,558 doses for Cohorts 1, 2, and 3 respectively. It is worth noting that with only one participant per group, these figures reflect individual experiences and cannot be generalised. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03344003 · results posted 9 August 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT03344003) looked at a treatment called Wilate® for people with moderate or severe Haemophilia A who had developed "inhibitors" — meaning their immune system was blocking the clotting factor medicine they needed. The trial had two groups: a retrospective cohort (looking back at past records) with 8 participants, and a prospective cohort (following people going forward) with 6 participants. The main thing being measured was whether a process called Immune Tolerance Induction (ITI) — a treatment approach aimed at getting the immune system to stop blocking the clotting factor — could be achieved fully or partially. Of the 14 people who started, 12 completed the study (6 in each group), and 2 in the retrospective group did not complete it. The reported data shows that, for the primary outcome, 7 participants in the retrospective cohort met the criteria for what the trial defined as complete ITI success (inhibitor levels dropping very low, clotting factor recovery returning to normal range, and the medicine lasting long enough in the body). One participant in the retrospective cohort met the criteria for "partial failure" (none of the three success criteria met, but inhibitor levels had fallen below a set threshold). No participants in the prospective cohort were recorded as achieving any level of success or failure under the reported categories. For all of the secondary outcomes — including how long it took to reach success, how long that success lasted, bleeding frequency, and use of additional medicines — the reported data shows no measurements were provided, which the trial notes was due to the study being terminated early. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02937831 · results posted 22 July 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT02937831) involved 6 participants, all of whom received a treatment called Nonacog Gamma, also known as Rixubis — a clotting factor product designed for people with haemophilia B (a condition where the blood does not clot properly due to low levels of a protein called Factor IX). Of the 6 participants who started the study, 2 completed it and 4 did not finish. The trial was measuring things like whether participants stopped using the treatment, whether their bodies developed a resistance to it (called an "inhibitor"), how often they had bleeding episodes, and how well the treatment managed bleeds or supported surgery. The reported data shows the following numbers across the primary outcome measures: 1 participant discontinued use of the treatment, and 0 participants developed a Factor IX inhibitor (meaning none of the participants' bodies appeared to resist the treatment in this way, though the small number of participants should be kept in mind). The reported annual bleed rate — the estimated number of bleeds per year — was 0. For participants on an "on-demand" regimen (meaning treatment was given when a bleed occurred rather than on a regular schedule), the data shows 1 dose was used to treat a bleed, and that 1 participant's bleed response was rated "Excellent" on a four-point scale, with 0 rated Good, Fair, or Poor. For the surgery-related outcomes, no measurement data was reported. It is worth noting that with only 6 participants enrolled and just 2 completing the study, the numbers reported here represent a very small group of people. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02695160 · results posted 19 July 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT02695160) enrolled only one participant, who received a high-dose infusion of an investigational gene therapy called SB-FIX. The trial was studying a treatment for haemophilia B, a condition where the body does not produce enough of a clotting protein called Factor IX. The trial was measuring things like unwanted medical events (called adverse events), changes in Factor IX protein levels in the blood, bleeding episodes, immune responses, and whether any of the therapy's genetic material could be detected in the bloodstream. The single participant did not complete the study. The reported data shows that one adverse event considered related to the treatment was recorded for the one participant. For the secondary outcomes, the reported change in Factor IX protein levels at 28 weeks was very small — a rise of 0.003 IU/mL for one measure and a slight fall of 0.006 IU/mL for another (IU/mL is simply a unit used to measure how much of a protein is present in the blood). Regarding Factor IX replacement therapy (standard clotting treatment the participant was already using), the reported data shows 170 uses before the study, 45 during the study period after receiving SB-FIX, and 125 at another recorded point — though the specific timeframes for each figure were not fully detailed in the submitted data. One bleeding episode was recorded. No immune response to Factor IX (measured as neutralising antibodies) was detected, reported as zero. The reported data also shows that genetic material from the therapy was detected in the participant's blood plasma. It is worth noting that with only one participant enrolled and that participant not completing the study, the reported figures are extremely limited in scope. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04431726 · results posted 14 June 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04431726) enrolled 55 participants, all of whom received at least one dose of the study drug, emicizumab, and all 55 completed 52 weeks in the study. The trial was measuring how often participants experienced bleeds while taking emicizumab, specifically looking at the rate of bleeds that required treatment and the rate of all bleeds over the course of a year. This type of rate is called an "annualised bleeding rate" (ABR) — simply put, it estimates how many bleeds a person would be expected to have in a 12-month period. The reported data shows that for bleeds which required treatment with a clotting medication, the estimated yearly rate was 0.4 bleeds per year using one calculation method (a statistical model), 0.5 bleeds per year using a direct average across participants, and 0.0 bleeds per year as the middle-point figure (meaning at least half of participants reported no treated bleeds during the study period). When looking at all bleeds — whether treated with medication or not — the reported data shows a rate of 2.0 bleeds per year using both the statistical model and the direct average, and 1.0 bleed per year as the middle-point figure. It is worth noting that all 55 participants are listed as "not completed" in the trial records, which the data indicates is because they moved into a longer-term follow-up phase rather than finishing the study outright; no participants are recorded as having fully completed the trial at the time this data was submitted. No comparison group (such as a placebo or alternative treatment group) was included in this trial, so the reported numbers reflect only the emicizumab group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04323098 · results posted 4 April 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 22 adults with severe haemophilia A (a condition where the blood does not clot properly due to very low levels of a protein called Factor VIII, or FVIII). All 22 participants received a single infusion of the gene therapy valoctocogene roxaparvovec (also called BMN 270). The trial was primarily measuring how much participants' FVIII activity levels changed from before the infusion to 52 weeks (about one year) afterwards. It also tracked how much clotting medication participants used, how often bleeding episodes occurred, and how participants rated their quality of life. The reported data shows that, on average, participants' FVIII activity levels rose by approximately 15 IU/dL (a unit used to measure clotting protein activity in the blood) from the starting baseline by Week 52. For context, the baseline was set at 1 IU/dL, reflecting the near-absent FVIII levels typical of severe haemophilia A. The reported data also shows changes in secondary measures over the follow-up period: the annualised (yearly) use of clotting factor replacement medication decreased by an average of about 4,150 IU/kg/year compared to baseline. The reported number of all bleeding episodes per year decreased by an average of about 6 bleeds per year, and bleeding episodes that required treatment with clotting factor decreased by an average of about 4 per year. On a quality-of-life questionnaire scored from 0 to 100 (where higher means better), the total score increased by an average of 6.7 points, and the physical functioning section increased by an average of 6.4 points, at Week 52. It is worth noting that while all 22 participants started the study and reached the Week 52 visit, the data shows zero participants were recorded as having formally "completed" the trial, suggesting the study was still ongoing at the time these results were submitted. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03861273 · results posted 27 March 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT03861273) enrolled 51 people, with 45 going on to receive the study treatment — a gene therapy called PF-06838435. The trial was measuring bleeding rates and other related outcomes in people with haemophilia B (a condition where the blood does not clot properly). Before receiving the gene therapy, participants had been tracked for at least six months while on their usual preventive treatment (regular infusions of a clotting protein called Factor IX), giving researchers a comparison point from each person's own history. The reported data shows that, looking at the period from Week 12 through to Month 15 after the gene therapy was given, the average number of total bleeds per year was 1.30 for participants on PF-06838435, compared with 4.43 per year during their earlier period on standard preventive infusions. For bleeds that required treatment, the reported figures were 0.73 bleeds per year after the gene therapy versus 3.35 per year beforehand. The reported data also shows that the average number of separate clotting-factor infusions per year dropped from about 59 (during standard treatment) to about 4 (after gene therapy), and the total amount of clotting factor used per year fell from roughly 3,171 units per kilogram of body weight to about 235 units per kilogram. Levels of circulating Factor IX — the clotting protein the body was now being prompted to produce — were also measured; the reported data shows these varied depending on which laboratory method was used, ranging from around 12–14% of normal up to around 26–28% of normal across different measurement approaches and time points. It is worth noting that 43 participants entered the long-term follow-up phase (Years 2–6), but no results from that phase appear in the data submitted to ClinicalTrials.gov at this time, so outcomes beyond Month 15 are not reported here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03734588 · results posted 23 February 2024

  According to the results reported on ClinicalTrials.gov, this trial investigated a treatment called SPK-8016 and enrolled 4 participants, all of whom completed the study. The trial was measuring a range of things related to how the body responded to the treatment, including whether participants experienced any adverse events (unwanted medical occurrences), whether there were any liver enzyme elevations requiring additional medication, levels of a clotting protein called Factor VIII (FVIII) in the blood, the number of bleeding episodes that occurred, and how often participants needed infusions. The reported data shows that all 4 participants experienced at least one adverse event during the study. No participants required immunosuppression (medications that calm the immune system) due to liver enzyme elevations. The reported data shows 6 spontaneous bleeding events and 1 traumatic bleeding event occurred across the group from 28 days after receiving the treatment. For the Factor VIII activity levels — both the peak (highest point) and steady-state (ongoing average) readings — and the annualised infusion rate (the estimated yearly number of infusions needed), the data was not reported in the structured results submitted to ClinicalTrials.gov. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04759131 · results posted 13 February 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04759131) enrolled 74 children with haemophilia A who were treated with a medicine called BIVV001. Thirty-eight children were under 6 years old and 36 were aged 6 to under 12 years. The trial was measuring two main things: whether the children's immune systems developed "inhibitors" (antibodies that can block the medicine from working) and how often bleeding episodes occurred while on treatment. The reported data shows that for the primary outcome — inhibitor development — zero participants in either age group developed these blocking antibodies during the study. For bleeding episodes, the trial tracked what is called an "annualised bleeding rate," meaning the estimated average number of bleeding episodes per person per year. For bleeds that required treatment, the reported figures were approximately 0.48 episodes per year in the under-6 group and 1.33 episodes per year in the 6-to-under-12 group. When all bleeding episodes were counted (both treated and untreated), the reported figures were approximately 2.78 per year in the younger group and 2.85 per year in the older group. A separate sensitivity analysis of the same data reported slightly different figures for the older group (0.75 treated bleeds per year; 2.32 total bleeds per year), while the younger group's figures remained the same. The reported data also shows bleeding broken down by type — spontaneous (no clear cause), traumatic (a known cause), and unknown. The reported treated bleed rates by type were approximately 0.17 (spontaneous), 0.28 (traumatic), and 0.03 (unknown) episodes per year in the under-6 group, and 0.14 (spontaneous), 0.59 (traumatic), and 0.59 (unknown) episodes per year in the 6-to-under-12 group. Two participants in the younger group did not complete the study; the data does not report the reasons. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02141074 · results posted 29 December 2023

  According to the results reported on ClinicalTrials.gov, this trial studied a clotting medication called nonacog beta pegol, which is used in people with haemophilia B (a condition where the blood does not clot properly due to low levels of a protein called Factor IX). The trial was split into two phases: a "pre-prophylaxis" phase, in which 34 participants started and 31 completed, and a "prophylaxis" (regular preventive dosing) phase, in which 51 participants started and 41 completed. A key thing the trial was measuring was whether participants developed "inhibitory antibodies" — meaning the body's immune system producing proteins that could block the medication from working — at different points during the trial. The reported data shows that, for inhibitory antibodies, 2 out of 34 participants in the pre-prophylaxis group had developed them by 50 treatment days and that number remained at 2 by the end of the trial. In the prophylaxis group, 0 participants had developed inhibitory antibodies at 50 treatment days, rising to 2 participants by 100 treatment days and remaining at 2 by the end of the trial. Regarding other medical events recorded during the trial (called adverse events — meaning any unwanted medical occurrence noted while on treatment, not necessarily caused by the medication), the reported data shows that by the end of the trial the pre-prophylaxis group had a total of 134 such events and the prophylaxis group had 794. The number of serious adverse events (those involving hospitalisation, life-threatening situations, or other significant medical concerns) by the end of the trial was 14 in the pre-prophylaxis group and 30 in the prophylaxis group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03370913 · results posted 28 November 2023

  According to the results reported on ClinicalTrials.gov, this trial enrolled 134 adults who received a single infusion of valoctocogene roxaparvovec (also known as BMN 270), a gene therapy being investigated for severe haemophilia A — a condition where the blood does not clot properly due to very low levels of a clotting protein called Factor VIII (FVIII). Of the 134 participants who started the trial, 132 completed it. The trial measured things like how often bleeds occurred, how much clotting factor medicine participants used, how their FVIII levels changed, and how their quality of life changed over roughly two years. The reported data shows that, on average, the number of all bleeding episodes per year changed by minus 4.13 bleeds per year compared to before the infusion, and the number of bleeds that needed clotting factor treatment changed by minus 4.08 bleeds per year. The amount of clotting factor medicine participants used per year changed by minus 3,891 units per kilogram of body weight per year compared to baseline. FVIII activity levels — a measure of how much clotting protein is present in the blood — showed a reported change of approximately 22 IU/dL (a standard unit for measuring clotting protein) from baseline at the two-year mark. The reported data also shows changes in participants' quality of life, as measured by a haemophilia-specific questionnaire scored on a scale of 0 to 100 (where higher scores mean better quality of life). The overall quality-of-life total score changed by plus 7.01 points from baseline at two years, and the physical functioning part of that score changed by plus 4.90 points. These figures describe what was measured and recorded; they do not on their own tell us how meaningful these changes were for individuals. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04878731 · results posted 7 July 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04878731) enrolled 6 participants, all of whom completed the study. Every participant received a single 300 mg dose of marstacimab (also called PF-06741086), given as an injection under the skin. The trial was primarily measuring how often participants experienced unwanted medical events (called adverse events) after receiving the study drug, as well as tracking certain blood test results over time. The reported data shows that 1 out of 6 participants experienced a treatment-emergent adverse event (that is, a new medical occurrence that appeared after receiving the study drug). No participants were reported to have experienced a serious adverse event, no one had a severe or life-threatening adverse event (graded 3, 4, or 5 on a standard medical scale), and no one stopped the study early because of an adverse event. For laboratory (blood and urine) test results, 6 out of 6 participants had at least one abnormal result noted, 1 participant had a result reported as abnormal in a specific category, and 2 participants had results in another category — though the data does not provide a more detailed breakdown of what those abnormalities were. The reported data also shows blood clotting measurements (called INR, a ratio used to assess how blood clots) taken at different time points, with values ranging from approximately 0.960 to 1.000 across the group, though individual time-point details were not fully labelled in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04085458 · results posted 28 June 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04085458) enrolled 32 people with severe haemophilia A — a condition where the blood does not clot properly. All participants received a clotting factor treatment called damoctocog alfa pegol (also known as Jivi or BAY94-9027). The trial was primarily measuring whether participants developed "inhibitors" — which are antibodies (proteins made by the immune system) that can block the clotting treatment from working. Twenty-seven of the 32 participants completed the trial, and five did not complete it. The reported data shows that, for the primary measure, zero out of 32 participants tested positive for these inhibitors during the trial. For the secondary measures, the reported data shows that 21 participants experienced at least one "treatment-emergent adverse event" — meaning an unwanted health event that occurred after starting the treatment; 3 participants had a serious adverse event; and 2 participants had an adverse event considered related to the study drug. The reported data also shows that 3 participants developed antibodies against a component of the treatment called PEG (a chemical attached to the medicine). The annualised bleeding rate — that is, the estimated average number of bleeds per person per year — was reported as 1.8 bleeds per year across the group. It is worth noting that some of the secondary outcome numbers appear in the data without full labels for each individual category, so not all breakdowns can be described in complete detail from the information provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04161495 · results posted 24 May 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04161495) enrolled a total of 159 participants across two groups. Group A ("Arm A") had 133 participants who received the study treatment, BIVV001, as a regular preventive (prophylaxis) regimen from the start. Group B ("Arm B") had 26 participants who first used the treatment on-demand (only when a bleed occurred) and then switched to a preventive regimen. The trial was primarily measuring the annualized bleeding rate — that is, the average estimated number of bleeding episodes that needed treatment per person over the course of a year. The reported data shows that for participants in Arm A on the preventive regimen, the reported average number of treated bleeding episodes per person per year was 0.71. For the secondary comparisons, the trial also looked at how Arm A participants' bleeding rates during the study compared to their bleeding rates recorded in an earlier observational study (used as a historical reference point). The reported data shows the historical reference group had an average of approximately 2.95–2.99 treated bleeding episodes per person per year, while the same participants' rate during the BIVV001 preventive regimen in this trial was reported as approximately 0.68–0.69 episodes per person per year. These figures were reported both as directly observed numbers and as estimates produced by a statistical model fitted to the data, and both approaches gave very similar results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03549871 · results posted 6 February 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03549871) enrolled 80 people with haemophilia across two groups: 30 participants who had developed inhibitors (antibodies that block standard clotting treatments, making those treatments less effective) and 50 who had not. The trial was measuring how often participants experienced bleeding episodes — expressed as an annualised bleeding rate (ABR), meaning the estimated number of bleeds per person per year. All participants first went through a period of up to 168 days on their usual clotting factor or bypassing agent therapy, before switching to the investigational treatment fitusiran for up to around 190 days. The reported data shows that during the standard therapy period, the overall observed bleeding rate across participants was approximately 7.56 bleeds per person per year, with a statistically modelled ("estimated") figure of 7.48. During the fitusiran treatment period, the reported observed rate was approximately 3.19 bleeds per person per year, with the modelled estimate at 2.91. For bleeds that happened without any clear cause (called spontaneous bleeds), the reported observed rates were approximately 5.09 per person per year during standard therapy and 2.51 during the fitusiran period. For joint bleeds specifically, the reported observed rates were approximately 5.35 per person per year during standard therapy and 2.82 during the fitusiran period. These are the numbers as submitted — the trial was not designed as a direct head-to-head comparison between two groups receiving different treatments at the same time, so these figures reflect a before-and-after measurement within the same participants. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04158648 · results posted 15 December 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT04158648) enrolled 73 participants, all of whom received the study drug emicizumab. The trial had one group only — there was no comparison group. The main thing the trial was measuring was how often participants experienced bleeds that needed treatment, tracked over time and expressed as an estimated number of treated bleeds per year (called an "annualised bleed rate"). Secondary measurements looked at all bleeds — whether treated or not — over the same period. The reported data shows that, for bleeds requiring treatment, the estimated average (using a statistical model) was 0.9 treated bleeds per year across the group. When calculated directly from each participant's own data, the group average was also 0.9 treated bleeds per year, while the midpoint figure — meaning half of participants were above this number and half were below — was 0.0 treated bleeds per year, indicating that more than half of participants reported no treated bleeds during the study period. For all bleeds combined (treated and untreated), the reported data shows an estimated rate of 2.3 bleeds per year using both the statistical model and the direct group average, with a midpoint figure of 1.0 bleed per year. It is worth noting that the data shows 73 participants started the study but zero were recorded as having formally "completed" it in the structured data — though 57 participants are noted as having reached 52 weeks in the study. The reasons for this are not explained in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02053792 · results posted 14 July 2022

  According to the results reported on ClinicalTrials.gov, this trial enrolled 97 people in total across two groups: 83 previously treated patients (people who had received Factor IX treatment before, called PTPs) and 14 previously untreated patients (people who had never received Factor IX treatment before, called PUPs). The trial was studying a clotting factor replacement product called CSL654 (also known as rIX-FP) for people with haemophilia B, a condition where the blood does not clot properly. The trial measured whether participants developed antibodies (called inhibitors) that could block the treatment, how well the product raised Factor IX levels in the blood, how often participants bled while on a regular prevention (prophylaxis) regimen, and how much of the product was used each month. The reported data shows that among the 83 previously treated patients, zero developed inhibitors against Factor IX, while one of the 14 previously untreated patients did. For the previously untreated patients, the reported data shows that a 50 IU/kg dose raised Factor IX blood levels by approximately 1.295 and 1.231 (IU/dL)/(IU/kg) — this is a measure of how much the blood level of the clotting factor rose for each unit of the product given per kilogram of body weight. For the previously treated patients on a regular prevention schedule, the reported annual bleeding rates (the estimated average number of bleeds per person per year) ranged from approximately 1.19 to 2.89 across different dosing regimens, with spontaneous (unprompted) bleeding rates ranging from approximately 0.60 to 1.32. The reported data shows that average monthly product use was around 181.8 IU/kg for previously treated patients and 188.53 IU/kg for previously untreated patients. Regarding adverse events (unwanted health events that occurred during the trial), the reported data shows that approximately 89.2% of previously treated patients and 91.7% of previously untreated patients experienced at least one adverse event during the trial; of those, approximately 2.4% of previously treated patients and 16.7% of previously untreated patients had at least one adverse event considered potentially related to the study product. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03489291 · results posted 16 June 2022

  According to the results reported on ClinicalTrials.gov, this trial involved 3 participants, all of whom completed the study. The trial was testing a single dose of a gene therapy called AMT-061 (also known as CSL222) in people with haemophilia B — a condition where the body does not produce enough of a clotting protein called Factor IX. The main thing the trial was measuring was whether that single dose could raise participants' Factor IX levels to at least 5% of normal by six weeks after the dose was given. The reported data shows that the average Factor IX activity level at the six-week primary measurement point was 30.6%, and a separate secondary measurement recorded an average of 40.8%. All 3 participants were reported to have stopped needing ongoing (continuous) Factor IX replacement therapy after receiving the gene therapy dose. The reported data also shows an annualised bleeding rate — that is, the estimated number of bleeds per person per year — with figures of 0.14, 0.07, 0.07, and 0.00 recorded across different time points or participants (the data as submitted does not clearly distinguish which figure applies to which). The annualised use of injected Factor IX was reported at 7.1 international units per kilogram per year overall, and 342.1 international units per year during the period after continuous prophylaxis ended. It is worth noting that this was a very small study of only 3 people, and the results as reported on ClinicalTrials.gov describe what was measured in those individuals only. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03417245 · results posted 4 February 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT03417245) enrolled 120 people in total — 40 in a group who used clotting factor medication only when a bleed occurred (called "on-demand" treatment), and 80 in a group who received monthly injections of fitusiran 80 mg as a preventive (prophylaxis) treatment. The trial was primarily measuring the "annualised bleeding rate" — that is, how many treated bleeding episodes each person would be expected to have over the course of a year. By the end of the study, 37 people in the on-demand group and 79 in the fitusiran group had completed the trial. The reported data shows that, for the main outcome during the core measurement period, the estimated number of treated bleeds per person per year was approximately 31.0 in the on-demand group and approximately 3.1 in the fitusiran group. A second way of looking at the same primary outcome — using straightforward observed counts rather than a statistical model — recorded approximately 21.8 treated bleeds per person per year in the on-demand group and 0.0 in the fitusiran group. For secondary outcomes covering the full treatment period (including the first four weeks of fitusiran use), the estimated figures were approximately 31.4 and 4.1 bleeds per person per year respectively, while the directly observed figures were approximately 25.2 and 1.8. When looking only at bleeds that happened for no clear reason (called spontaneous bleeds), the estimated rate during the core period was approximately 22.0 per person per year in the on-demand group and approximately 1.8 in the fitusiran group; the directly observed figures were approximately 16.1 and 0.0 respectively. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03417102 · results posted 21 December 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03417102) enrolled people with haemophilia A or B who have developed antibodies (called "inhibitors") that make standard clotting factor treatments less effective. A total of 19 participants were assigned to an on-demand group, meaning they used existing medicines called bypassing agents only when a bleed occurred, while 38 participants were assigned to receive monthly injections of a medicine called fitusiran (80 mg) as a preventive (prophylactic) treatment. All 19 in the on-demand group completed the study, while 33 of the 38 in the fitusiran group completed it. The main thing the trial was measuring was the annualised bleeding rate — that is, how many treated bleeding episodes each person experienced on average over the course of a year. The reported data shows that, in the on-demand group, the observed average was approximately 18.1 treated bleeds per person per year during the main measurement period. In the fitusiran prophylaxis group, the reported observed average was approximately 1.7 treated bleeds per person per year over the same period. For bleeds that happened for no obvious reason (called spontaneous bleeds), the reported data shows an observed average of approximately 15.6 per person per year in the on-demand group, compared with approximately 0.9 per person per year in the fitusiran group. Similar figures were also reported when measured across the full treatment period. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03103542 · results posted 30 November 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03103542) enrolled 16 people, all of whom received a clotting factor treatment called recombinant Factor VIII Fc (rFVIIIFc). The trial was looking at whether this treatment could help people with haemophilia A who had developed "inhibitors" — antibodies that block clotting factor from working properly. This process is called Immune Tolerance Induction (ITI), and the main goal was to see how many participants could reach a defined level of success, measured by their inhibitor levels dropping, their clotting factor recovering properly, and the treatment lasting long enough in the body. Of the 16 who started, 9 completed the study and 7 did not. The reported data shows that, for the primary goal of ITI success, the numbers reported across measurement time points were 1, 2, 6, and 7 participants meeting the success criteria (the data as submitted lists four separate measurements without clearly labelling which time point each belongs to). For those who did reach the success criteria, the reported average time to do so was approximately 46.71 weeks, with a related figure of 38.76 weeks also reported. The reported data shows that 0 participants experienced a relapse — defined as inhibitor levels rising again — during the 48-week follow-up period after ITI success. The reported annualised bleeding rate (the estimated number of bleeds per year requiring treatment) was 4.70 during ITI treatment and 5.07 during the follow-up period. The reported data also includes adverse event (unwanted health events) counts across several categories, with 188 total adverse events, 21 serious adverse events, and 170 treatment-emergent adverse events (those occurring after treatment began) reported among the 16 participants; additional subcategory figures were also submitted. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03528551 · results posted 26 November 2021

  According to the results reported on ClinicalTrials.gov, this trial looked at a treatment called N8-GP (a clotting factor replacement) in people with haemophilia. A total of 160 participants were enrolled and split into three groups based on how often they received the treatment: 25 people received it once a week, 133 received it twice a week, and 2 received it three times a week. The trial ran for up to 104 weeks (about two years) and measured things like unwanted health events, bleeding episodes, and how well bleeds were controlled when treated. The reported data shows that across the study period, the number of recorded adverse events (unexpected or unwanted health occurrences that happened while on the treatment) was 58 in the once-weekly group, 444 in the twice-weekly group, and 8 in the three-times-weekly group. Regarding bleeding, the total number of bleeding episodes recorded were 123, 190, and 14 across the three groups respectively. Of those, spontaneous bleeds (ones with no obvious cause) numbered 98, 73, and 8. When bleeds were treated with N8-GP, the reported data shows that in the once-weekly group 114 bleeding episodes were rated "excellent" in response and 8 were rated "good"; in the twice-weekly group 94 were rated "excellent", 80 "good", and 4 "moderate"; and in the three-times-weekly group 8 were rated "excellent" and 6 "good". The reported data also shows that on average, participants needed approximately 1.2, 1.5, and 1.1 injections per bleeding episode in each group respectively. Notably, no participants in any group were reported to have developed antibodies against the clotting factor (known as inhibitors), which can sometimes interfere with treatment. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02172950 · results posted 27 October 2021

  According to the results reported on ClinicalTrials.gov, this trial looked at a clotting factor treatment called CSL627 in people with haemophilia A — a condition where the blood does not clot properly. The trial enrolled two groups: 222 people who had previously been treated for haemophilia (called "Previously Treated Patients" or PTPs), and 24 people who had never been treated before ("Previously Untreated Patients" or PUPs). Of those who started, 197 PTPs and 19 PUPs completed the study. The trial was mainly measuring whether participants developed "inhibitors" — antibodies that can block the treatment from working — as well as how often bleeding occurred and how well bleeding episodes were controlled. The reported data shows that among the previously treated patients, 0% developed inhibitors after 100 days of exposure to CSL627. For the previously untreated patients, 5 out of those who reached at least 50 days of exposure developed high-level inhibitors (defined as a level of 5 or more Bethesda units per millilitre, a standard laboratory measurement). For major bleeding episodes in PUPs, 100% were rated as successfully treated by the treating doctor. The reported annual rate of spontaneous (unprovoked) bleeds in PUPs was 1.9 for those on a regular preventive dosing schedule and 4.04 for those treated only when bleeding occurred. For previously treated patients, 87.1% of all bleeding episodes were rated as successfully treated. The reported annual overall bleeding rates were also recorded — for PTPs these were 28.32 (on-demand) and 2.84 (preventive dosing), and for PUPs 5.12 (on-demand) and 5.94 (preventive dosing). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03196284 · results posted 22 October 2021

  According to the results reported on ClinicalTrials.gov, this trial involved people with a bleeding disorder who were assigned to receive either concizumab (a investigational injectable medicine) or eptacog alfa (a comparator treatment). The main part of the trial included 17 people in the concizumab group and 9 people in the eptacog alfa group, with all 17 concizumab participants and 8 of the 9 eptacog alfa participants completing that phase. A further 25 participants then took part in an extension phase, all receiving concizumab for a longer period. The trial was primarily measuring the number of bleeding episodes that were treated over time. The reported data shows that during the main part of the trial (at least 24 weeks), the concizumab group recorded 47 treated bleeding episodes in total, while the eptacog alfa group recorded 77 treated bleeding episodes. When looking only at bleeds that happened without a clear cause (called spontaneous bleeds) during this same period, the concizumab groups recorded 19 episodes (at the lower dose) and 5 episodes (at the higher dose), compared with 69 episodes in the eptacog alfa group. Over the longer extension period of at least 76 weeks, the reported data shows a combined total of 256 treated bleeding episodes across all concizumab participants, and spontaneous bleeds of 36, 9, and 7 episodes across the three different dose levels used. The reported data also shows the number of medical events that occurred during the trial that were not necessarily related to the treatment. During the main part, there were 39 such events recorded in concizumab participants at the lower dose, 4 at the higher dose, and 18 in the eptacog alfa group. Over the full 76-week period, concizumab participants recorded 104, 24, and 3 such events across the three dose levels respectively. These numbers are counts of events as recorded, and the trial was not designed to establish whether any particular event was caused by the treatment. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02962765 · results posted 28 September 2021

  According to the results reported on ClinicalTrials.gov, 78 people took part in this trial of a clotting factor replacement product called Nuwiq® (also known as Human-cl rhFVIII), which is used in people with haemophilia A (a condition where the blood does not clot properly). The trial was measuring two main things: whether participants developed antibodies against the treatment (called "inhibitors," which can stop the treatment from working) and whether participants experienced any unwanted reactions linked to the treatment. Most participants (77 out of 78) were on a preventive dosing schedule, while a small number (2 participants) received the treatment only when a bleed occurred. Sixty-one participants completed the trial, and 17 did not. The reported data shows that, for both primary outcomes, the number recorded was zero — meaning no participants were reported to have developed inhibitors at any point during the trial (at the start, during, or at the end), and no adverse drug reactions were recorded in the study population. For the secondary outcomes, the reported data shows that participants on the preventive schedule experienced an average of 2.39 bleeding episodes per year. When bleeding episodes were rated on a four-point scale (excellent, good, moderate, or none) in the preventive group, 167 episodes were rated "excellent," 50 "good," 26 "moderate," and 3 "none." In the small on-demand group, 50 bleeding episodes were rated "excellent," 4 "good," 1 "moderate," and 0 "none." For the 4 participants who had surgery during the trial, all 5 surgical procedures were rated "excellent" by the treating doctors, with none rated good, moderate, or none. The reported data shows results from a single group with no comparison group, which is worth keeping in mind when reading these figures. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03407651 · results posted 23 September 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03407651) looked at a medicine called MarzAA in people with a bleeding disorder. The trial was run in three parts. In the first part (Part 1a), 11 people started and 10 completed it. In the second part (Part 1b), 9 people started and all 9 completed it. In the third part (Part 2), 11 people started and 8 completed it, with 3 not finishing. Overall, the trial was measuring how often participants experienced bleeding episodes while taking MarzAA, compared to how often they had bled in the past. It also tracked certain blood-clotting measurements and whether any serious clotting events occurred. The reported data shows that the main outcome — a score called the annualised bleed rate (ABR), which is simply a count of how many bleeding episodes a person would be expected to have in a year — was reported as 1.46 during Part 2 of the trial. For context, the trial's starting assumption was that participants would have at least 12 bleeds per year (roughly one per month) without preventive treatment, and a lower ABR score reflects fewer bleeding events. On this measure, scores can range from 0 (no bleeds) to 365. For the secondary outcomes, the reported data shows that breakthrough bleeds (bleeds needing a higher dose) numbered zero in Parts 1a and 1b, five in the Part 2 group receiving the 30 µg/kg dose, and zero in the Part 2 group receiving the 60 µg/kg dose. The number of clinical thrombotic events (blood clots forming where they shouldn't) was reported as zero across all groups. The trial also tracked changes in several blood-clotting lab measurements from before each dose; these figures were reported but the data as submitted does not include enough detail to describe the full range of individual results beyond the summary change values provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04286412 · results posted 30 July 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT04286412) enrolled 25 participants, all of whom completed the study. All 25 participants received a clotting medicine called Nonacog Alfa, which is a manufactured version of Factor IX — a protein the blood needs to clot properly. The main thing the trial was measuring was whether participants developed antibodies (called "inhibitors") against the medicine, which can reduce how well it works. The trial also tracked bleeding episodes, how much medicine was used, and any unwanted health events that occurred. The reported data shows that none of the 25 participants (0%) developed Factor IX inhibitors during the study. On the safety-related measures, none of the participants experienced a serious adverse event or a medically important event — which in this trial included things like blood clots or severe allergic reactions. Three out of 25 participants did experience what are called "treatment-emergent adverse events," meaning any health event that occurred after starting the medicine, regardless of whether it was thought to be related to the medicine. The reported average annual amount of medicine used per person was approximately 224,582 international units (IU) — a standard way of measuring clotting medicines — or roughly 3,639 IU per kilogram of body weight per year. The data for the average annual bleeding rate was not reported in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03363321 · results posted 27 July 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03363321) enrolled a total of 20 people across five groups, each receiving different dosing regimens of the study drug. The groups varied by dose amount and whether participants were also taking certain other medications (called "inhibitors") that can affect how the body processes drugs. The trial was primarily measuring participants' physical responses to the treatment — specifically, whether they experienced any unwanted medical events, unusual blood or urine test results, changes in vital signs (such as blood pressure or heart rate), changes in heart rhythm readings (ECGs), notable physical examination findings, or reactions at the injection site. The reported data shows that across the five groups, the number of participants who experienced any unwanted medical event (called a treatment-emergent adverse event, or TEAE) ranged from 1 to 5 people per group. Of the two groups where the data was clearly reported, 2 participants in one group and 1 in another experienced events considered more serious or severe. Regarding abnormal blood or urine test results, the reported data shows small numbers — generally 0 to 1 person per group met the pre-defined thresholds for concern. For vital signs, only 1 participant across all groups met the criteria for a notable change. For heart rhythm readings, no participants in the one group where this was measured met any of the pre-defined criteria for concern. The reported data shows no injection site reactions were recorded in any group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01811875 · results posted 22 July 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT01811875) involved 7 people who took part, with 5 completing the study and 2 not completing it. All participants received a clotting factor treatment called Optivate (a Factor VIII concentrate, a protein used to help blood clot). The trial was looking at whether participants developed something called "inhibitors" — which are antibodies the body can produce that work against the clotting factor treatment — as well as how well the treatment was absorbed into the bloodstream and how often breakthrough bleeds (unexpected bleeding episodes) occurred during the roughly 12-month study period. The reported data shows that all 5 participants who completed the study did not develop inhibitors to Factor VIII (meaning their test results stayed below the threshold of 0.6 Bethesda Units, the cut-off used to define inhibitor development). For the absorption (called "recovery") measurements — which tracked how much of the clotting factor actually entered the bloodstream after a dose — the reported figures showed a very small difference of –0.91 IU/dL per IU/kg when comparing participants' previous treatment to their first dose of Optivate, and an adjusted average difference of –0.01 IU/dL per IU/kg across the four main study visits. The reported data also shows that participants experienced an average of approximately 3.99 breakthrough bleeds per person per year, were exposed to the treatment on an average of about 116 days per person per year, and received an average total preventive (prophylactic) dose of around 3,640 IU/kg per person over the study period. It is worth noting that with only 7 participants enrolled and 5 completing the study, this was a very small trial, so the numbers above reflect a very limited group of people. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT04072237 · results posted 16 July 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT04072237) looked at how a drug called MarzAA moves through the body at different doses and delivery methods. A total of 75 participants took part across nine stages. In the first stage, 11 people received MarzAA through a drip into a vein (intravenous, or IV) at a dose of 18 micrograms per kilogram of body weight. In the remaining eight stages, groups of 8 people each received MarzAA as an injection under the skin (subcutaneous, or SC) at increasing doses — ranging from 30 up to three separate doses of 60 micrograms per kilogram given three hours apart. The trial was primarily measuring how much of the drug was present in the blood over time (a concept called "area under the curve," or AUC — essentially a way of tracking the total amount of drug the body was exposed to). The reported data shows that the total drug exposure in the blood (AUC) generally increased as doses went up. For the intravenous group (Stage 1), the reported AUC figure was 1,390 h·ng/mL. For the under-the-skin injection groups, the reported AUC figures ranged from around 516 h·ng/mL at the lowest dose (Stage 2, 30 µg/kg) up to approximately 3,236 h·ng/mL at the highest dosing stage (Stage 9, three doses of 60 µg/kg). The reported data also shows that when the AUC figure was adjusted to account for the different dose sizes, the values were broadly similar across all the under-the-skin injection stages (roughly 14 to 17 h·kg/mL), suggesting a fairly consistent relationship between dose and exposure for those groups. For the secondary measurements, the reported data shows that the peak level of MarzAA detected in the blood (Cmax) was much higher in the intravenous group (about 419 ng/mL) than in any of the under-the-skin groups (ranging from about 19 to 98 ng/mL). The time it took to reach that peak level (Tmax) was very short for the IV group (around 0.17 hours, i.e. roughly 10 minutes), compared to around 6.75 to 12.25 hours for the under-the-skin groups. The reported data also includes figures for how long the drug remained in the body (a measure called "half-life") — for the IV group this was reported as approximately 3.3 hours, while for the under-the-skin groups it ranged from about 13 to 19 hours; a related early half-life measure was only reported for the IV group, and was listed as not available for all other stages. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03191799 · results posted 11 June 2021

  According to the results reported on ClinicalTrials.gov, this trial enrolled 195 people with haemophilia A who were given a weekly injection of emicizumab (at a dose of 1.5 mg/kg). Of those, 193 received at least one dose, and 186 completed the study. Nine participants did not complete it. The trial was primarily focused on tracking unwanted medical events (called adverse events) that participants experienced, as well as monitoring things like blood test results and vital signs such as blood pressure and body temperature. The reported data shows that 163 out of 193 participants who received the drug experienced at least one adverse event of any kind. Serious adverse events — meaning those that were considered medically significant regardless of severity — were recorded in 39 participants. Grade 3 or higher adverse events (rated as severe or worse on a standard scale) occurred in 31 participants. One participant died. When expressed as a rate, there were about 212 adverse events per 100 patient-years for all types combined, about 13 per 100 patient-years for serious events, and about 17 per 100 patient-years for severe or worse events. The reported data also shows that changes in vital signs — body temperature, systolic blood pressure, and diastolic blood pressure — were very small across all measured time points during the study, though the data does not allow a direct conclusion to be drawn from these numbers alone. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03361137 · results posted 1 March 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03361137) enrolled 14 people in total who had haemophilia A — a condition where the blood does not clot properly. Participants were already receiving a medicine called emicizumab and were divided into groups based on whether they had a complication called "inhibitors" (which makes standard treatment harder) and the type of procedure they were having: removal of a central venous access device (a tube inserted into a large vein), a simple dental extraction, or — for one participant — a surgery that ultimately was not performed. The trial was measuring how much bleeding occurred around the time of these procedures, whether extra clotting medicines were needed, and what the level of emicizumab in the blood was on the day of the procedure. The reported data shows that, among participants who had a CVAD removal or dental extraction, the percentage who did not have excessive bleeding and did not need extra clotting medicine ranged from 66.7% to 100% across the different groups. For the CVAD removal group with inhibitors, 66.7% met this measure, while all participants (100%) in the dental extraction group with inhibitors, and both other surgery groups without inhibitors, met it. Separately, the reported data shows that 11.1% of participants in the CVAD removal group with inhibitors did experience excessive bleeding requiring extra clotting medicine, while 0% did in the remaining groups. After going home from their procedure, 22.2% of the CVAD removal group with inhibitors reported any bleeding requiring extra treatment, and notably 100% of that same group reported using extra clotting medicine after discharge — though the very small numbers in each group (as few as one or two people) mean these percentages should be read with caution. The reported emicizumab concentration in the blood on the day of surgery was around 53.74 micrograms per millilitre for participants with inhibitors and 38.40 micrograms per millilitre for those without. Regarding surgical complications needing hospitalisation or a return to surgery, 0% was reported across all groups that completed a procedure. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03597022 · results posted 30 November 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03597022) tested three different doses of an investigational drug called BAY1093884 — 100 mg, 225 mg, and 400 mg. A total of 24 people took part, with 8 in each dose group. Notably, none of the participants completed the study — all 24 did not finish, though the reasons for this are not detailed in the reported data. The trial was primarily measuring safety-related events, specifically looking at unwanted medical occurrences (called adverse events) that happened during or shortly after treatment. The reported data shows that when it came to unwanted medical events that investigators considered possibly linked to the study drug, 1 out of 8 participants in the 100 mg group, 4 out of 8 in the 225 mg group, and 5 out of 8 in the 400 mg group experienced such events. For more serious adverse events — those involving hospitalisation, life-threatening situations, or significant disability — the reported numbers were 1, 2, and 1 participants across the three dose groups respectively. Of those serious events, the investigators considered 0, 2, and 1 to be possibly related to the study drug. The trial also tracked specific events of concern such as blood clotting or allergic reactions: 0 participants in the 100 mg group, 2 in the 225 mg group, and 1 in the 400 mg group experienced these. No participants in any group were reported to have clinically significant abnormal laboratory test results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03186677 · results posted 10 November 2020

  According to the results reported on ClinicalTrials.gov, a total of 16 people took part in this trial across five groups (called cohorts). The first nine participants were enrolled in an earlier phase (Period 1, three people per cohort), and the remaining seven joined a later phase (Period 2, with five in one cohort and two in another). All 16 participants completed the trial without dropping out. The trial was measuring things related to an investigational product — tracking unwanted events that occurred after it was given, how much of the active substance reached the bloodstream, and whether the body produced certain proteins (called neutralising antibodies) that could work against the treatment. The reported data shows that the number of unwanted events (called adverse events — unexpected or undesirable things that happened during the trial) varied considerably between groups: Cohort 1 recorded 0 adverse events, Cohorts 2 and 3 each recorded 10, Cohort 4 recorded 69, and Cohort 5 recorded 47. For the measure of how much active substance reached the bloodstream (called Cmax, or peak blood level), the reported figures — expressed as a percentage of normal clotting activity — were 71.10% for Cohort 1, 100.80% for Cohort 2, 41.70% for Cohort 3, 15.34% for Cohort 4, and 23.80% for Cohort 5. A second set of Cmax figures was also reported for Cohorts 1, 2, and 3 (70.47%, 5.30%, and 5.27% respectively), though the data was not reported for Cohorts 4 and 5 for this second measurement. Regarding neutralising antibodies — proteins the body can make that may interfere with a treatment — the reported data shows that no participants in Cohorts 1, 2, 3, or 4 had these detected at the end of the study, while 2 out of 2 participants in Cohort 5 did have them detected. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01311648 · results posted 3 November 2020

  According to the results reported on ClinicalTrials.gov, this trial enrolled children aged 0 to 12 years with haemophilia A and looked at a clotting factor treatment given as regular preventive (prophylaxis) infusions. There were two broad groups: 51 children who had been treated with clotting factor before (called PTPs, aged 0–12 years) and 43 children who were either new to treatment or minimally treated (called PUPs/MTPs, aged under 6 years). The trial had a main study phase and an extension phase. The main thing being measured was how many bleeding episodes occurred within 48 hours of each preventive infusion, expressed as an annualised (per-year) figure. The reported data shows that, for the annualised number of bleeds occurring within 48 hours of a preventive infusion, the average (mean) figures were approximately 2.2 bleeds per year for previously treated children aged 0–under 6, about 1.9 bleeds per year for previously treated children aged 6–12, and about 1.9 bleeds per year for the newer-to-treatment group. When looking at the middle value (median) rather than the average, the figures were roughly 1.9, 0.0, and 0.0 bleeds per year respectively for the same three groups. For the broader measure of all bleeds during the entire prophylaxis period, the reported averages ranged from about 3.4 to 7.1 bleeds per year depending on the group. Regarding a specific immune response called an "inhibitor" — where the body develops antibodies that can reduce how well the treatment works — the reported data shows that none of the 51 previously treated children developed inhibitors during the main study, while 6 of the newer-to-treatment children developed low-level inhibitors and 17 developed high-level inhibitors. Only 3 surgeries were recorded across all groups during the study, so the data on bleeding control during surgery is very limited. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02234323 · results posted 13 August 2020

  According to the results reported on ClinicalTrials.gov, this trial enrolled 108 children with haemophilia A — a condition where the blood does not clot properly due to low levels of a protein called Factor VIII. The trial tested a treatment called recombinant Factor VIII Fc fusion protein (rFVIIIFc), a manufactured version of that clotting protein. Of the 108 who enrolled, 103 went on to receive the treatment. Participants were placed into one of three treatment approaches: an "on-demand" (episodic) regimen where the treatment was given when a bleed occurred (81 participants), a preventive (prophylactic) regimen given regularly to try to prevent bleeds (89 participants), and an immune tolerance induction (ITI) regimen for participants who had developed antibodies against Factor VIII treatment (15 participants). Some participants moved between regimens during the study. The trial's main goal was to measure how many children developed "inhibitors" — antibodies that can block the treatment from working. The reported data shows that the primary outcome — confirmed inhibitor development — was recorded in approximately 31% of participants who had received at least 10 treatment days and had at least one inhibitor test performed. For the secondary outcomes, the reported average number of bleeding episodes per person per year (called the annualised bleeding rate, or ABR) was 2.24 for the episodic group, 1.49 for the prophylactic group, and 0.00 for the ITI group. The reported average number of spontaneous joint bleeds per person per year was 0.00 across all three groups. When parents or caregivers rated how each injection responded to a bleed using a four-point scale, the reported data shows the majority of rated responses fell into the "excellent" or "good" categories across all regimens, though the specific breakdown figures across each regimen and rating category are listed in the full data. The reported average annual dose of the treatment was approximately 198 IU/kg per person per year for the episodic group, around 5,384 IU/kg for the prophylactic group, and approximately 67,310 IU/kg for the ITI group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02234310 · results posted 31 July 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT02234310) enrolled 33 children with haemophilia B (a condition where the blood does not clot properly due to low levels of a protein called Factor IX). All 33 participants received a treatment called recombinant Factor IX Fc fusion protein (rFIXFc). Some children were placed on a schedule where they only received injections when a bleed occurred (22 participants), while others received regular preventive injections (28 participants — noting some participants switched between approaches during the study). A total of 27 participants completed the trial. The main thing the trial was measuring was whether participants developed "inhibitors" — that is, whether their immune system produced antibodies that blocked the treatment from working. The reported data shows that, for the primary outcome, approximately 3% of participants (1 out of 33) developed confirmed inhibitors, as detected by a specialised laboratory test. For the secondary outcomes, the reported average number of bleeding episodes per person per year was 0.21 for those on the preventive schedule and 1.24 for those on the on-demand (treat-when-bleeding) schedule. The reported average number of spontaneous joint bleeds (bleeds into joints with no known cause) per person per year was 0.00 for both groups. When treatment responses to injections for bleeding episodes were rated by parents or carers on a four-point scale, the reported data shows the majority of rated responses fell into the "excellent" or "good" categories, though the full breakdown across both treatment approaches varied. The reported average total amount of the study medicine used per person per year was approximately 203 IU per kilogram for the preventive group and approximately 3,175 IU per kilogram for the on-demand group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00947193 · results posted 16 June 2020

  According to the results reported on ClinicalTrials.gov, this trial tested a medicine called ataluren in people with haemophilia — a condition where the blood does not clot properly. Participants received one of two different dose levels of ataluren over 14 days. A total of 13 people were enrolled: 3 in the lower-dose group and 10 in the higher-dose group. Notably, all 3 participants in the lower-dose group did not complete the study, while all 10 in the higher-dose group did. The main thing the trial was trying to measure was whether, after 14 days, participants had a blood clotting factor activity level reach at least 1% — a threshold that was set as the target for a meaningful response. The reported data shows that, for the primary measure, none (0 out of 10) of the participants in the higher-dose group reached that clotting factor activity threshold by Day 14. For one of the secondary measures — looking at whether any participants developed certain antibodies in their blood that could interfere with clotting factors — 1 out of 10 participants showed a change from their starting level. Regarding unwanted events (side effects) experienced during the study, the reported data shows that 8 out of 13 participants across both groups experienced at least one adverse event (an unwanted health occurrence during the trial), and 2 experienced a serious adverse event. No Grade 3 (severe) or Grade 4 (life-threatening) adverse events were reported. For blood and urine test abnormalities, 1 participant had a result flagged as clinically meaningful in one category, and none in the others. Blood levels of ataluren were also measured, with figures of 14.7 and 6.66 micrograms per millilitre recorded before and two hours after a morning dose, respectively. Compliance data was not collected because participants left the study early. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03204539 · results posted 29 May 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03204539) was set up to compare two approaches — an "Alternative Treatment" and a "Standard Treatment" — in people with a clotting condition who had developed inhibitors (antibodies that block treatment). The trial was designed to measure things like how quickly the inhibitors disappeared, whether treatment fully or partially succeeded, bleeding episodes during treatment, quality of life, and costs. The two groups were called "Alternative Treatment" and "Standard Treatment." The reported data shows that only one participant was enrolled into the Standard Treatment group, and zero participants were enrolled into the Alternative Treatment group. That single participant did not complete the study. Because enrollment was so low, no measurement results were recorded for any of the outcome measures — not for the primary outcome (time to negative inhibitor) nor for any of the six secondary outcomes, including bleeding events, quality of life scores, or treatment costs. The data was not reported for any of these measures. In plain terms, the trial appears to have stopped very early after enrolling just one person, meaning there are essentially no findings to describe from this study. No conclusions about either treatment approach can be drawn from the numbers submitted. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02343406 · results posted 22 May 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT02343406) enrolled 266 participants across several groups. The adult part of the trial compared a drug called ABT-414 (also known as depatuxizumab mafodotin) — given either alone or combined with temozolomide — against a control group receiving standard chemotherapy (temozolomide or lomustine) in adults with brain cancer. A small separate group of 6 children also took part. The trial measured how long adult participants lived overall (called "overall survival"), how long they lived without their disease getting worse (called "progression-free survival"), and in the children's group, how the drug moved through the body and what side effects were recorded. Very few participants formally "completed" the study — most left before the scheduled end, which is common in trials involving serious illness. The reported data shows that for overall survival in adults, the median time (meaning the point at which half the participants had died and half had not) was 9.6 months for the ABT-414 plus temozolomide group, 7.9 months for the ABT-414 alone group, and 8.2 months for the control group. For progression-free survival — how long before the disease got worse — the reported medians were 2.7 months for ABT-414 plus temozolomide, 1.9 months for ABT-414 alone, and 1.9 months for the control group. In the children's group, 100% of the 6 participants reported at least one adverse event (an unwanted health change recorded during the study). The reported peak level of ABT-414 in the blood of the children was 31.4 µg/mL, and the drug was reported to take approximately 9 days to reduce to half its peak level in the body. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02484092 · results posted 19 May 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT02484092) enrolled 15 participants, all of whom completed the study. All 15 received a single intravenous infusion of SPK-9001, a gene therapy being investigated for haemophilia B. The trial was measuring a range of safety-related outcomes, including changes in physical examination findings, vital signs, blood and urine test results, any unwanted medical events that occurred after the infusion (called treatment-emergent adverse events, or TEAEs), immune responses to the gene therapy vector, and whether any participant's clotting factor activity reached an unexpectedly high level. The reported data shows that, out of 15 participants, zero had clinically significant changes in their physical examination findings, and zero had clinically significant changes in vital signs (such as blood pressure, heart rate, or temperature). Two participants had abnormal laboratory results (blood or urine tests) that were recorded as TEAEs, and two participants experienced TEAEs that were considered related to the treatment. The reported data shows that no participants experienced a serious adverse event (such as hospitalisation or a life-threatening event) that was considered treatment-related. Two participants showed a positive immune response to the gene therapy vector (meaning their immune system appeared to react to it), and no participants reached the unexpectedly high clotting factor activity level that was being monitored for safety reasons. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01480180 · results posted 20 April 2020

  According to the results reported on ClinicalTrials.gov, this trial tested a clotting factor treatment called N8-GP in people with haemophilia A (a condition where the blood does not clot properly). The trial ran in several phases over a period of roughly 80 months (about six and a half years). In the main phase, 174 people were assigned to a regular preventive dosing schedule (called prophylaxis) and 12 to an on-demand approach (taking the treatment only when a bleed occurred). Participants then continued into extension phases, where different dosing schedules were compared. The trial was measuring two main things: how often participants developed antibodies (called inhibitors) that could block the treatment from working, and how many bleeding episodes participants experienced per year on the preventive schedule. The reported data shows that, when it came to inhibitor development — that is, the body producing antibodies against the treatment — the rate was very low across the groups studied. At approximately 19 months, 25 months, and again at around 80 months, the reported inhibitor rate for the preventive dosing groups was 0.006 (meaning roughly 6 in every 1,000 eligible participants), while the on-demand groups reported a rate of 0 across all time points. For the number of bleeds per participant per year, the reported data shows: on the every-four-day preventive schedule, participants had approximately 1.33 bleeds per year at 19 months, 1.36 at 25 months, and 0.99 at 80 months. On the every-seven-day preventive schedule, the reported figures were 0 bleeds per year at 25 months and 1.95 bleeds per year at 80 months. Bleeding rate data for the on-demand group was not reported as a primary outcome. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03449342 · results posted 18 March 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03449342) enrolled 60 people in total — 10 adolescents aged 12 to under 18, and 50 adults aged 18 and over. All 60 participants completed the study with no drop-outs. The trial was investigating a clotting factor treatment called turoctocog alfa, used for a bleeding disorder called haemophilia A. The main thing the trial was set up to measure was whether participants developed antibodies (called "inhibitors") against the treatment — a known complication where the body's immune system can block the treatment from working. Secondary measurements included side effects, allergic reactions, how many bleeding episodes were rated as having a successful response, and how much of the treatment was used over the course of a year. The reported data shows that none of the 60 participants — neither the adolescents nor the adults — developed these antibodies (inhibitors) at or above the threshold measured (0.6 BU) during the 8-week treatment period. The reported data also shows that no adverse drug reactions, serious adverse drug reactions, or allergic/infusion reactions linked to the treatment were recorded in either age group (reported as zero reactions per patient-year). For bleeding episodes, 2 episodes in the adolescent group and 38 episodes in the adult group were reported as having a "successful haemostatic effect" — meaning the response was rated as either excellent or good within approximately 8 hours. The reported annualised consumption of the treatment was approximately 7,030 IU per kilogram of body weight per year for adolescents, and approximately 6,086 IU per kilogram of body weight per year for adults. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01489111 · results posted 21 January 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT01489111) enrolled 36 people, all of whom received the investigational treatment turoctocog alfa pegol (also called N8-GP), a clotting factor product being studied in people with haemophilia A (a condition where the blood does not clot properly). Thirty-five of the 36 participants completed the study, and one did not. The trial was measuring how well the treatment controlled bleeding during and after surgery, using a four-point rating scale: "excellent," "good," "moderate," or "none." The reported data shows that, for the primary outcome — bleeding control rated by the surgeon right at the end of the operation — out of 49 surgeries assessed, 25 were rated "excellent," 22 were rated "good," 2 were rated "moderate," and none were rated "none." For the days immediately after surgery (days 1–6), the reported data shows 0 bleeding episodes rated "excellent," 1 rated "good," 0 rated "moderate," and 0 rated "none," with 1 episode where the rating data was not fully specified in the structured results. For the later post-operative period (days 7–14), the reported data shows 1 episode rated "excellent," 1 rated "good," and none rated "moderate" or "none." The average amount of the treatment used during surgery was reported as 20.7 IU/kg (a measure of dose per kilogram of body weight), and 33.0 IU/kg across days 1–6 after surgery. The reported data also shows that zero participants developed antibodies (called inhibitors) against the clotting factor during the trial — inhibitors are proteins the body can sometimes produce that may reduce a treatment's ability to work. No comparison group was included in this trial, so all results reflect a single group of participants receiving the one treatment. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03376516 · results posted 24 December 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT03376516) enrolled 11 people, of whom 10 received the study treatment — a clotting factor product called Wilate — and 9 completed the study. The trial was designed to measure how Wilate moves through the body over time in people with haemophilia A (a condition where the blood does not clot properly). This type of measurement is called pharmacokinetics, or PK for short — it looks at things like how quickly the treatment reaches its peak level in the blood, how long it stays there, and how the body processes it over time. The reported data shows the following PK measurements for Wilate, each presented as a minimum value, maximum value, and an average (middle) figure across the 10 participants: The peak level of the clotting factor in the blood (maximum plasma concentration) was reported as roughly 79 to 83 units per decilitre of blood, with an average of about 81. The time it took to reach that peak level was reported as 0.25 hours (about 15 minutes) across all measurements. The "half-life" — meaning the time it took for the level in the blood to fall to half its peak — was reported as between approximately 8.3 and 9.4 hours, averaging around 8.8 hours. The overall exposure to the treatment over time (called AUC) was reported as between approximately 672 and 769 units·hours per decilitre, with an average of about 720. The average time the treatment spent in the body overall (mean residence time) was reported as between about 11.5 and 12.6 hours, averaging around 12 hours. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01992549 · results posted 17 December 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT01992549) enrolled 48 children with haemophilia A (a condition where the blood does not clot properly) who were treated with a clotting factor medicine called Human-cl rhFVIII. The main thing the trial was measuring was whether participants developed "inhibitors" — that is, antibodies that the body can produce against clotting factor treatments, which can make those treatments less useful. The trial also tracked how often spontaneous bleeds occurred, how well the medicine performed when used to treat bleeds or during surgery, and how many participants experienced any unwanted health events (adverse events). Forty-four of the 48 participants completed the study. The reported data shows that, for the primary outcome, zero out of 48 participants developed inhibitors during the observation period. For spontaneous breakthrough bleeds during preventive (prophylactic) treatment, the reported average rate was approximately 0.28 bleeding episodes per year. When it came to treating actual bleeding episodes (reported across 29 participants), the study recorded 58 bleeding episodes in total; of these, 28 were rated "excellent," 21 "good," 3 "moderate," and 1 "none" by parents/guardians and investigators. For surgical use, the data covered a very small number of procedures (2 minor and 2 major surgeries reported across 3 participants), with ratings not fully detailed in the submitted data. Regarding unwanted health events, the reported data shows 29 out of 48 participants experienced at least one adverse event; 5 were reported as possibly related to the study medicine, 3 were described as serious, and 1 participant withdrew due to an adverse event. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01085344 · results posted 5 December 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 56 participants, all of whom received Factor VIII (a clotting protein used by people with haemophilia). Fifty of the 56 participants completed the study, while 6 did not finish. The trial was measuring things like joint bleeding, how much of the clotting product participants used, and whether any participants developed antibodies (called "inhibitors") that could interfere with the treatment. The reported data shows that 17 out of 56 participants developed what the study called "target joint bleeding" — defined as three or more bleeds into the same joint within a three-month period. On average, participants experienced approximately 0.95 joint bleeds per person per year. The reported average annual use of Factor VIII was 3,600 IU/kg (a measure of the dose used per kilogram of body weight, per year). Five participants were reported to have developed an inhibitor — meaning their body produced antibodies that could work against the clotting product. Regarding physical ability, the reported data shows a median score of 0 out of 3 on a disability questionnaire (where 0 means no disability and 3 means maximum disability). For joint health assessed by a physiotherapy scoring tool, the reported median end-of-study scores across the joints measured were either 0 or 1, on scales ranging from 0 (no joint damage) to 26 or 30 (maximum possible damage depending on the joint). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02974855 · results posted 4 December 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02974855) enrolled 26 people in total across four groups, each receiving different doses or dosing schedules of an investigational medicine called PF-06741086, given as an injection under the skin. Some participants had a condition called an "inhibitor" (meaning their body had developed antibodies that reduced the effectiveness of standard clotting treatment), while others did not. The trial was primarily measuring unexpected medical events that occurred during treatment — known as treatment-emergent adverse events, or TEAEs — as well as changes in certain blood and urine test results. The reported data shows that across all 26 participants, 21 experienced at least one TEAE during the study. Of those, 14 were considered treatment-related by the investigating doctors. Two participants (one from the loading-dose non-inhibitor group and one from the inhibitor group) left the study early due to a TEAE, and both of those were also considered treatment-related. The reported data shows that no participants in any group met the pre-specified thresholds for abnormal results in blood count tests, blood chemistry tests, or urine tests. For a protein in the blood called globulin, small changes from starting levels were reported across the groups, ranging from a slight decrease to a very slight increase, though the clinical significance of these changes is not described in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02256917 · results posted 3 December 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02256917) involved 58 participants, all of whom received a treatment called Human-cl rhFVIII, a clotting factor product. The trial was studying people with haemophilia A — a condition where the blood does not clot properly — and was measuring how often bleeding events occurred when participants received a personalised (individually tailored) preventive dosing schedule, compared to figures from a separate historical group of patients who had only taken the treatment when a bleed actually happened (known as "on-demand" treatment). The reported data shows that participants on the individually tailored preventive schedule had a reported average of approximately 4.67 total bleeding events per year, compared to a historical on-demand figure of around 58.08 bleeding events per year. For spontaneous bleeds (those happening without an obvious cause), the reported figures were approximately 2.98 events per year on the preventive schedule, versus around 38.46 events per year in the historical on-demand group. The reported data also shows that participants who received the preventive treatment twice a week or less had an average of approximately 4.82 total bleeding events per year. The typical gap between doses was reported as around 83–84 hours (roughly three and a half days). These comparisons are between two separate groups of patients — not a head-to-head randomised comparison — so they should be understood in that context. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03205163 · results posted 2 December 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT03205163) enrolled 16 adult participants in total — 7 in a lower-dose group and 9 in a higher-dose group. Each participant received two treatments in sequence: first a single dose of Advate (a standard factor VIII replacement product), and then a single dose of BIVV001 (an investigational factor VIII product). The trial was measuring things like unwanted medical events (called adverse events), abnormal laboratory test results, whether participants developed antibodies that could interfere with the treatment (known as "inhibitors"), and how the body processed each medicine over time. The reported data shows that during the Advate period, 2 out of 7 lower-dose participants and 1 out of 9 higher-dose participants experienced adverse events; 1 lower-dose participant had a serious adverse event and none in the higher-dose group did. During the BIVV001 period, 3 out of 6 lower-dose participants and 6 out of 9 higher-dose participants experienced adverse events; 1 lower-dose participant had a serious adverse event and none in the higher-dose group did. No participants in either group showed clinically significant abnormal laboratory results during either treatment period. Regarding inhibitor development — where the body produces antibodies that could counteract the treatment — the reported rate was 0% in both the lower- and higher-dose groups. For one secondary measure looking at how high the product's level in the blood peaked (called "maximum concentration"), the reported figures for the lower-dose group were 51.8 IU/dL for Advate and 70.1 IU/dL for BIVV001; results for the higher-dose comparison were not reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01731600 · results posted 30 October 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 68 children with haemophilia A (a condition where the blood does not clot properly), split into two age groups: 34 younger children aged 0–5 years, and 34 older children aged 6–11 years. All 34 older children completed the trial, while 28 of the 34 younger children completed it (6 did not finish). The trial was measuring whether a clotting factor treatment called N8-GP led to the development of "inhibitors" — proteins the body can produce that block the treatment from working — and also tracked bleeding rates, how well bleeds were controlled, and how much of the medicine was used. The reported data shows that in the main part of the trial, zero participants in either age group developed inhibitory antibodies (the blocking proteins) at or above the threshold of 0.6 Bethesda units. Regarding bleeding episodes, the reported number of bleeds per patient per year during the main trial was approximately 1.94 for younger children and 1.97 for older children; over the full trial period these figures were reported as 0.61 and 0.93 respectively. When bleeds were treated, the average number of injections needed was between 1.5 and 1.9 depending on age group and trial phase. The reported rate of all adverse events (unwanted health occurrences, whether serious or not) was around 4.87 per patient-year for younger children and 4.74 for older children in the main trial, dropping to 3.09 and 2.45 respectively across the full trial period. The haemostatic effect data (how well bleeds were controlled, rated as excellent, good, moderate, or none) was reported as a count of bleeding episodes in each category, though a full breakdown across all categories was not clearly separated in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02548143 · results posted 6 September 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02548143) looked at a treatment called LR769 in people with haemophilia (a condition where the blood does not clot properly) who were undergoing surgery. Twelve people took part in total — six in a "major surgery" group and six in a "minor surgery" group. Five people in each group completed the trial, with one person in each group not finishing. The trial was measuring how well bleeding was controlled around the time of surgery, with researchers rating each procedure as "excellent," "good," or something less than good, based on how much blood was lost compared to what would be expected in a person without a bleeding disorder. The reported data shows that for the primary outcome — the investigator's overall rating of each procedure — four of the major surgery procedures were rated "excellent" and two were rated "good." For the minor surgery group, five procedures were rated "excellent" and none were rated "good." For the secondary outcomes, which used a similar rating approach, the reported data shows that across two separate assessment points: in one assessment, four major surgery procedures and six minor surgery procedures were rated as "good" or "excellent" combined, with no procedures falling outside that range in either group; in another assessment, five major surgery procedures and two minor surgery procedures were rated as "good" or "excellent" combined, again with none falling outside that range. It is worth noting that the total numbers of procedures assessed may differ slightly from the number of participants, as some individuals may have undergone more than one procedure. The reported data shows only small numbers of participants and procedures across both groups, which means these figures give a limited picture. No data was reported for outcomes such as longer-term follow-up or other measures beyond the ratings described above. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02585960 · results posted 26 August 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02585960) looked at a treatment called BAX 855 in people with haemophilia A (a condition where the blood does not clot properly). A total of 121 participants were enrolled across three groups: 57 in a lower-dose group, 58 in a higher-dose group, and 6 in a separate non-randomised group. The trial measured how often participants experienced bleeds over time, with a particular focus on the second six months of the study period. The reported data shows that the main thing being measured — the percentage of participants who had zero bleeds in the second six-month period — was 42.1% in the lower-dose group and 62.1% in the higher-dose group. For the secondary measures, the reported average number of bleeds per year in the second six months was approximately 3.6 for the lower-dose group and 1.6 for the higher-dose group. When looking only at bleeds that happened without an obvious injury (called spontaneous bleeds), the reported rates were approximately 2.5 per year for the lower-dose group and 0.7 per year for the higher-dose group. Bleeds related to an injury were reported at approximately 1.1 per year and 0.9 per year respectively. Bleeds specifically into joints were reported at approximately 2.6 per year in the lower-dose group and 1.1 per year in the higher-dose group. The total amount of medication used per kilogram of body weight was also recorded, with the lower-dose group using approximately 3,985 international units per kilogram and the higher-dose group using approximately 7,031 international units per kilogram over the study period. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01741545 · results posted 24 June 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 51 people across two groups — 12 participants in Cohort A and 39 in Cohort B. The trial was measuring how the body responded to treatment for hepatitis C (HCV), a virus that affects the liver. The main thing being tracked was whether the virus became undetectable in participants' blood 12 weeks after finishing treatment — a result researchers call "SVR12" (sustained virologic response at 12 weeks). A number of additional measures were also tracked at different points during and after treatment. The reported data shows that for the main outcome (virus undetectable at 12 weeks after treatment), 91.7% of Cohort A participants and 89.7% of Cohort B participants reached that point. For the additional measures: at week 4 of treatment, the virus was undetectable in 91.7% of Cohort A and 76.9% of Cohort B; at week 12 of treatment, those figures were 91.7% and 92.3% respectively; and by the end of the full treatment course, 100% of participants in both groups had undetectable virus levels. At 24 weeks after finishing treatment, the reported figures were lower — 66.7% for Cohort A and 30.8% for Cohort B. The reported data also shows that 0% of participants in either group experienced certain blood abnormalities (low red blood cells, low white blood cells, or low platelets) during treatment. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03248141 · results posted 20 May 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT03248141) enrolled participants with haemophilia — a condition where the blood does not clot properly. There were two groups: one for people with Haemophilia A and one for people with Haemophilia B. In total, 11 people joined the Haemophilia A group, and no one joined the Haemophilia B group. All 11 participants in the Haemophilia A group completed the study, and none dropped out. The trial was set up to look at how participants used healthcare resources (such as hospital visits or treatments) and how they were dosed with their medication over the course of the study. The reported data shows that, despite the trial having two primary outcome measures — "Resource Utilisation Pattern" and "Dosing Pattern" — no numerical results were submitted for either of these outcomes on ClinicalTrials.gov. In other words, while the study was completed by all 11 participants, the actual measurement data for what the trial was designed to track has not been reported in the structured results available. No figures are available to describe what was found for either group. Because no outcome numbers have been provided in the publicly available results, it is not possible to describe what the trial found about resource use or dosing patterns. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01945593 · results posted 1 May 2019

  According to the results reported on ClinicalTrials.gov, this trial looked at a treatment called BAX 855, which is a type of Factor VIII (a clotting protein used to manage haemophilia A). A total of 218 people took part across four age groups: 3 children under 2 years old, 64 children aged 2 to under 12, 26 teenagers aged 12 to under 17, and 125 adults aged 17 and over. The trial was measuring, among other things, whether participants developed antibodies (called inhibitors) that could work against the treatment, how often spontaneous bleeds (bleeds that happen without an obvious injury) occurred per year, and how well the treatment managed breakthrough bleeding episodes when they did occur. The reported data shows that across all four age groups, zero participants developed inhibitory antibodies to Factor VIII during the study. For spontaneous bleeds, the reported data shows varying bleed rates depending on age group and dosing schedule, with figures ranging roughly from about 0.6 to 2.0 bleeds per year across the different groups and regimens — the full breakdown by dosing schedule was not straightforward to separate from the data as submitted. For total bleeds (both spontaneous and injury-related combined), the reported figures ranged from approximately 1.9 bleeds per year in the youngest group to around 3.2 bleeds per year in the 12-to-under-17 age group. When breakthrough bleeds were treated, participants and caregivers rated the response on a scale of excellent, good, fair, or none — the reported data shows the majority of treated bleeds received a rating of either excellent or good across all age groups. On average, participants needed approximately 1.3 to 1.4 infusions to treat each bleeding episode, and the average time between bleeding episodes was reported as roughly 5 to 5.8 months across the age groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01271868 · results posted 11 April 2019

  According to the results reported on ClinicalTrials.gov, this trial involved 9 participants, all of whom received the study treatment, called IB1001. All 9 participants completed both parts of the trial — a pharmacokinetic study (which looked at how the treatment moves through the body) and a treatment study (which looked at how many doses were needed to control bleeding episodes). The trial was measuring how the body processed IB1001 and how many infusions (doses given through a drip) were needed to stop bleeds. The reported data shows that, across the bleeding episodes treated, the number of infusions needed for bleed control varied: the recorded values were 4, 25, 1, 1, 1, and 1 infusions per episode. Regarding how the body handled IB1001, the reported data shows the substance reached a peak level of 51.7 IU/dL in the blood, and its "half-life" — meaning the time it took for the level in the blood to fall by half — was reported as 22.6 hours. The total exposure to the treatment over time (a measure called area under the curve) was reported as 1,062 IU·hr/dL, and the rate at which the body cleared the treatment was reported as 7.3 mL per kilogram per hour. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03034044 · results posted 5 April 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT03034044) enrolled 106 participants, all of whom were assigned to receive a medicine called Xyntha (also known as Xyntha Solofuse, a clotting factor treatment). Of those, 105 people received the medicine and completed the study; one person did not complete it. The trial was measuring a range of things related to unwanted medical events (called adverse events) that occurred during the study, as well as how well the medicine appeared to control bleeding when given before surgery. The reported data shows that, out of 105 participants who received Xyntha, 15 experienced at least one unwanted medical event (adverse event) during the study period, while no participants experienced a serious adverse event. Of those 15, 10 had events described as mild (not interfering with normal daily activities), 5 had events described as moderate (interfering to some extent with daily activities), and none had severe events. The reported data also shows that 2 participants had an adverse event considered by the investigators to be possibly related to the treatment, and no serious treatment-related adverse events were reported. No participants stopped the study because of an adverse event, and no participants died. For the small group of 6 participants who received the medicine as preparation for surgery, all 6 had their bleeding response rated as "excellent" — meaning clear pain relief or improvement in bleeding signs within 8 hours, with no additional infusion needed. No participants in that group were rated as good, moderate, or no response. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00543439 · results posted 11 January 2019

  According to the results reported on ClinicalTrials.gov, this trial tested a clotting factor medicine called moroctocog alfa (AF-CC) in people with haemophilia A (a condition where the blood does not clot properly). A total of 66 people were enrolled across three groups. One group started on "on-demand" treatment — meaning they only took the medicine when a bleed occurred — before switching to a regular preventive (called "routine prophylaxis") dose of 25 IU/kg. The other two groups were already on routine preventive treatment and were compared on two different doses: 25 IU/kg and 45 IU/kg. The trial tracked how many bleeds participants had per year, how well single infusions controlled bleeds, and other related measures. The reported data shows that participants in the on-demand group experienced an average of 47.0 bleeds per year while on that approach, compared to an average of 1.5 bleeds per year when they switched to the preventive 25 IU/kg dose. For the group already on preventive treatment, those receiving 45 IU/kg reported an average of 3.3 bleeds per year, while those on 25 IU/kg reported an average of 2.2 bleeds per year. When looking at how well a single infusion controlled a bleed across all participants, the average number of infusions needed per bleeding episode was reported as 1.5. Of 559 bleeds assessed for response to the first infusion during on-demand treatment, 376 were rated "excellent," 150 "good," 27 "moderate," and 2 showed "no response," with 4 not recorded. The reported data also shows that among participants on preventive treatment, a small number — 2 out of those on 45 IU/kg and 1 out of those on the 25 IU/kg group — needed their preventive dosing schedule stepped up to a more intensive level due to ongoing spontaneous bleeds. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02541942 · results posted 4 January 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 19 participants, and all 19 completed the study with no drop-outs. The trial was a genetic sub-study linked to an earlier clinical trial (called adept™2), which had tested a clotting treatment in people with haemophilia. The purpose of this sub-study was to look at two specific things in participants' DNA: their HLA type (a set of genetic markers related to the immune system, covering up to eight different categories) and whether they had any variations, known as polymorphisms, in the gene that controls a clotting protein called Factor VII. The reported data shows that, across the eight HLA categories tested, the number of participants carrying the most likely genetic variants ranged from as few as 1 participant to as many as 15 participants, depending on the specific category. For example, the largest single group had 15 participants sharing a particular HLA variant, while several categories had only 1 participant each. For the second measurement — variations in the Factor VII gene — the reported data shows that zero polymorphisms (that is, no gene variations of this type) were detected across the participants tested. The reported data does not include further breakdown of what these genetic findings mean in terms of health outcomes, and no additional outcome figures beyond those counts were submitted. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03020160 · results posted 26 December 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT03020160) enrolled 48 people in total across two groups: 7 participants in a smaller "pharmacokinetic run-in" group (used to study how the body processes the medicine, emicizumab) and 41 participants in the main "expansion" group. All 48 participants completed at least 24 weeks in the study. The trial was primarily measuring how often bleeds occurred per year — known as the "annualised bleeding rate" — in people receiving emicizumab, a medicine being studied for haemophilia A. A secondary measure looked at participants' self-reported quality of life using a standard questionnaire. The reported data shows that, in the main expansion group of 41 participants, the annualised rate of bleeds that required treatment was 2.4 per year. When all bleeds were counted (whether treated or not), the reported rate was 4.5 per year. Breaking this down further, the reported rate for treated bleeds that happened without an obvious cause (called spontaneous bleeds) was 0.6 per year; for treated joint bleeds it was 1.7 per year; and for treated bleeds in so-called "target joints" — joints that had bled repeatedly before the study — it was 1.0 per year. The reported data also shows a secondary quality-of-life finding. Adult participants completed a haemophilia-specific questionnaire (scored from 0 to 100, where lower scores reflect better quality of life) at the start and again at week 25. The reported average change in total score was −13.62 points, meaning scores were, on average, about 13.6 points lower at week 25 than at the start. The questionnaire's designers had defined a drop of 7 or more points as a "clinically meaningful" change, though what this means for any individual was not reported in the data provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01580293 · results posted 6 December 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT01580293) looked at a clotting treatment called BAY94-9027 for people with haemophilia A (a condition where the blood does not clot properly). The trial had two main parts. Part A enrolled 20 people who received treatment only when a bleed occurred ("on-demand"), and 114 people who received the treatment on a regular schedule to try to prevent bleeds ("prophylaxis"). Part A had a main phase and an extension phase. Part B enrolled 19 people who were having major surgery. The trial tracked how often bleeds occurred under different dosing schedules — some participants received injections twice a week, others every five days, and others every seven days. The reported data shows that in the main trial phase (Part A), the on-demand group had an annualised bleed rate — that is, an estimated number of bleeds per year — of about 23.4. For the various prophylaxis (preventive dosing) groups, the reported annualised bleed rates were considerably lower, ranging from approximately 1.9 to 4.1 bleeds per year depending on the dosing schedule. During the extension phase, the on-demand group's annualised bleed rate was reported at around 34.1, while the prophylaxis groups ranged from approximately 0.7 to 3.1 bleeds per year. The reported data also shows that zero participants in either the on-demand or prophylaxis groups developed antibodies (called inhibitors) against the treatment during Part A — inhibitors can reduce how well clotting treatments work. Data on bleed locations and how many injections were needed to control individual bleeds were also recorded, though no summary conclusions about those figures were reported beyond the raw counts. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01454739 · results posted 23 November 2018

  According to the results reported on ClinicalTrials.gov, this trial enrolled 240 children with haemophilia A (a condition where the blood does not clot properly). The children came from two groups: 61 from one earlier study and 179 from three other related studies. The trial was measuring whether a clotting factor treatment called rFVIIIFc caused the body to develop "inhibitors" — which are antibodies that can block the treatment from working — and it also tracked how often participants experienced bleeding episodes. The reported data shows that for the primary outcome — checking whether any child developed inhibitors — the result was zero out of all participants across both age groups (children under 6, and children aged 6 to under 12) and across all study groups. No positive inhibitor results were reported. For bleeding episodes, the reported data shows that children on regular preventive dosing ("tailored prophylaxis") had annualized bleeding rates of approximately 1.18 episodes per year (younger age group) and 1.59 episodes per year (older age group) in the dedicated paediatric study, while children in the combined studies group on tailored prophylaxis had a rate of around 0.64 episodes per year. Higher rates were reported among children who only received treatment when bleeding occurred rather than on a preventive schedule. For the doctors' overall assessment of how participants responded to the treatment, the reported data shows that across both groups the large majority of recorded assessments were rated "excellent" (403 out of 428 responses in one group, and 1,061 out of 1,252 in the other), with a small number rated "effective" or "partially effective", and none rated "ineffective." These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01425723 · results posted 23 November 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT01425723) involved 120 children with haemophilia B — 27 from one earlier study and 93 from another — who received a clotting factor treatment called rFIXFc. The trial was measuring whether children developed "inhibitors" (antibodies that can block a clotting treatment from working), how often bleeding episodes occurred, and several other related things such as how much of the treatment was used and how doctors rated participants' responses to the treatment. The reported data shows that, for the primary outcome — whether any child developed inhibitors — the number was zero across all groups, meaning no confirmed inhibitor development was recorded in any participant. For bleeding rates (the average number of bleeding episodes per person per year), the reported figures varied depending on the treatment schedule and age group: children under six years on a regular preventive schedule had a reported rate of around 1.04 episodes per year, those aged six to under twelve years had around 1.14 episodes per year, and participants from the other study group had a reported rate of around 2.26 episodes per year on a similar schedule, with higher rates recorded for those on an on-demand (treat-when-bleeding) schedule. For spontaneous joint bleeds specifically, rates of zero were reported for the younger children on preventive schedules, with small numbers reported for some other groups. Doctors' assessments of how participants responded to the treatment were recorded across hundreds of visits; the reported data shows the large majority of responses fell into the top two categories on the four-point scale, with very few in the lower two categories, and none recorded in the lowest category. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02618915 · results posted 14 November 2018

  According to the results reported on ClinicalTrials.gov, this trial involved a total of 6 people split into two small groups of 3 participants each — Cohort 1 and Cohort 2. All 6 participants completed the study with no dropouts. The trial was testing an investigational gene therapy called DTX101, intended for people with haemophilia B (a condition where the blood lacks enough of a clotting protein called Factor IX, or FIX). The study was measuring things like unwanted medical events (called adverse events), changes in the level of FIX activity in the blood, bleeding episodes, and use of replacement clotting therapy. The reported data shows that all 3 participants in Cohort 1 and all 3 in Cohort 2 experienced at least one adverse event (an unwanted medical occurrence during the study). One participant in Cohort 1 experienced a serious adverse event, while none in Cohort 2 did. One participant in Cohort 1 also experienced a treatment-related adverse event, compared to none in Cohort 2. Regarding FIX activity levels in the blood (measured in IU/dL, a standard unit for clotting factor), the reported change from the starting point at Week 6 was an average increase of 5.00 IU/dL in Cohort 1 and 9.80 IU/dL in Cohort 2. Changes in FIX activity were also tracked at multiple other time points, with figures varying across the weeks for both groups. The reported annualised bleeding rate (estimated number of bleeds per year) was 8.7 episodes per year for Cohort 1 and 5.0 for Cohort 2. Annualised use of replacement FIX therapy was reported as approximately 350,115 IU/kg per year for Cohort 1 and 64,247 IU/kg per year for Cohort 2. At no time point across either group were any neutralising antibodies against FIX detected in any participant. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02847637 · results posted 14 November 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT02847637) enrolled 152 people in total across four groups. Eighteen participants received no preventive treatment at first before later switching to emicizumab (the control group, Arm C); 36 received emicizumab at 1.5 mg/kg once a week (Arm A); 35 received emicizumab at 3 mg/kg every two weeks (Arm B); and 63 who had previously been on a standard clotting-factor treatment received emicizumab at 1.5 mg/kg once a week (Arm D). The trial was measuring how often participants experienced bleeds — specifically tracking bleeds that needed treatment, bleeds in joints, bleeds that happened without an obvious cause (spontaneous), and bleeds in joints that had been repeatedly affected before the trial started. The vast majority of participants completed the study. The reported data shows that the main outcome — the estimated number of treated bleeds per year — differed notably across groups. The control group (no preventive treatment) recorded an estimated 38.2 treated bleeds per year, while the three emicizumab groups recorded 1.5, 1.3, and 1.6 treated bleeds per year respectively. When all bleeds (treated and untreated) were counted, the control group recorded an estimated 47.6 per year, compared with 2.5, 2.6, and 3.3 per year in the emicizumab groups. For bleeds specifically in joints, the control group recorded an estimated 26.5 treated joint bleeds per year, versus 1.1, 0.9, and 1.2 in the emicizumab groups. For spontaneous treated bleeds, the figures were 15.6 per year in the control group and 1.0, 0.3, and 0.5 in the emicizumab groups. For bleeds in repeatedly affected ("target") joints, the control group recorded 13.0 per year versus 0.6, 0.7, and 0.6 in the emicizumab groups. A separate within-person comparison for participants who had previously been on standard clotting-factor treatment showed an estimated 4.8 treated bleeds per year before the study, compared with 1.5 per year while on emicizumab during the study. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01493778 · results posted 30 October 2018

  According to the results reported on ClinicalTrials.gov, this trial enrolled 60 children with haemophilia A — a condition where the blood does not clot properly due to low levels of a protein called Factor VIII. The trial tested a treatment called turoctocog alfa (also called N8), which is a manufactured version of Factor VIII. Participants either received the treatment regularly to try to prevent bleeds (preventive treatment) or only when a bleed occurred (on-demand treatment). Of the 60 who started, 52 completed the trial and 8 did not finish. The main thing the trial was measuring was how many participants developed "inhibitors" — antibodies that the body can produce against Factor VIII replacement treatments, which can make those treatments less useful. The reported data shows that in the preventive treatment group during the main phase of the trial, 43.1% of participants developed inhibitors at a level considered significant (0.6 or more Bethesda Units per millilitre — a standard way of measuring these antibodies). For bleeding rates, the reported data shows that participants on preventive treatment experienced an average of around 5.63 bleeds per patient per year during the main phase, dropping to 3.81 in the extension phase. Those on on-demand treatment had an average of 4.27 bleeds per patient per year during the main phase. On average, it took about 1.8 to 2.8 injections to stop a bleed, depending on which group and phase was measured. The reported data also shows that participants in the inhibitor group used considerably more of the treatment per year on average (around 21,783 IU/kg — a measure of dose per body weight) compared to those in the preventive treatment groups (around 4,317 to 5,723 IU/kg per year). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00768287 · results posted 10 September 2018

  According to the results reported on ClinicalTrials.gov, this trial involved people with haemophilia B (a condition where the blood does not clot properly due to low levels of a protein called Factor IX). The trial tested a treatment called IB1001, comparing how it behaved in the body versus an existing Factor IX product called Nonacog Alfa, and also looked at how well it managed bleeding episodes. In total, 32 people took part in the initial pharmacokinetics study (which measures how a drug moves through the body), 14 of those completed a repeat of that study, 67 people took part in the treatment phase (58 on a regular preventive schedule and 9 using the treatment only when bleeds occurred), and 17 people took part in a surgical sub-study. The reported data shows that, for the pharmacokinetics portion, IB1001 and Nonacog Alfa had broadly similar measurements. The time the drug remained active in the body (called "half-life" — meaning the time it takes for the level to drop by half) was reported as approximately 24.2 hours for IB1001 and 26.4 hours for Nonacog Alfa. Peak Factor IX levels reached after a dose were reported as 73.7 IU/dL for IB1001 and 72.8 IU/dL for Nonacog Alfa. Other technical measures of how the drug spread and was processed in the body were also similar between the two products, and the repeat IB1001 measurements were consistent with the first set of results. For the primary outcome — how well bleeding episodes were controlled — participants rated each bleed after treatment. The reported data shows that, across both the on-demand group (treating bleeds as they happened) and the prophylaxis group (regular preventive dosing), the majority of bleeding episodes were rated as "excellent" or "good." Specifically, in the on-demand group, 53 episodes were rated excellent and 105 good, while in the prophylaxis group, 116 were rated excellent and 74 good. Smaller numbers were rated "fair" (25 on-demand, 28 prophylaxis) or "poor" (3 on-demand, 9 prophylaxis), with some episodes not assessed. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01913405 · results posted 14 May 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT01913405) enrolled 22 people with haemophilia who were treated with a medicine called BAX855. The trial was measuring how well bleeding was controlled during and after surgery, using a scoring system called the Global Hemostatic Efficacy Assessment (GHEA). This score combined three separate ratings — one taken during surgery, one the day after surgery, and one covering the full period around surgery up to discharge or day 14 — to give an overall picture of how bleeding was managed compared to what would typically be expected in someone without a bleeding disorder. Twenty of the 22 participants completed the trial; two did not complete it, though the reason is not specified in the reported data. The reported data shows that across all surgery types — major orthopaedic (bone and joint), major non-orthopaedic, and minor surgery — 100% of surgeries in both the full analysis group and the per-protocol group received a GHEA score in the "Excellent" or "Good" range, meaning no surgery was rated "Fair" or below. For blood loss volumes, the reported median figures varied by surgery type. For major orthopaedic surgery, the overall perioperative (around the time of surgery) blood loss median was reported as 246 mL, while for major non-orthopaedic surgery it was 5.5 mL, and for minor surgery it was 9 mL. Blood transfusions were recorded for some participants — the reported median transfusion volume across the full group was 438 mL, though the reported data notes this figure only applies to those who actually received a transfusion. A small number of bleeding episodes were also recorded: 2 events in total across the full analysis group, with 1 each in the non-orthopaedic and minor surgery subgroups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02502149 · results posted 6 April 2018

  According to the results reported on ClinicalTrials.gov, this trial looked at how a clotting factor medicine called rFVIIIFc (a recombinant Factor VIII) behaved in the body after it was given as an injection. The trial was specifically comparing two different manufacturing scales of the same medicine — a smaller production batch (called "2K") and a larger production batch (called "15K") — to see whether the larger-scale product moved through the body in a similar way to the smaller-scale version. A total of 24 participants took part in the first comparison stage, and 23 of those went on to a further stage. Twenty-three participants then took part in the second stage, split across two different vial strengths of the 15K product. The reported data shows that the two main things being measured were: (1) the total exposure to the medicine over time (called AUCinf — essentially how much of the medicine was present in the blood and for how long), and (2) how much the medicine level in the blood rose per unit of dose given (called incremental recovery). For the smaller-batch (2K) product, the reported AUCinf figure was 2,255.6 IU·h/dL, and for the larger-batch (15K) product it was 2,425.8 IU·h/dL. The incremental recovery figures were 2.684 IU/dL per IU/kg for the 2K product and 2.700 IU/dL per IU/kg for the 15K product. The reported data also shows that the peak level of the medicine in the blood (Cmax) was 133.20 IU/dL for the 2K product and 134.67 IU/dL for the 15K product; the time for the medicine level to fall by half (half-life) was about 14.2 hours for 2K and 14.8 hours for 15K; the rate at which the body cleared the medicine was 2.18 mL/h/kg for 2K and 2.04 mL/h/kg for 15K; and the estimated volume in which the medicine distributed through the body was 43.80 mL/kg for 2K and 43.01 mL/kg for 15K. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01863758 · results posted 30 January 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT01863758) enrolled 66 people with severe haemophilia A — a condition where the blood does not clot properly. All 66 participants completed the early phases of the study, and 64 of the 66 completed the final phase. The trial was measuring how often bleeding episodes occurred when participants received a clotting factor product called Human-cl rhFVIII on a regular preventive (prophylactic) schedule, rather than only when a bleed happened. It also looked at how the dosing was spaced out over time and how much of the product was used each week. The reported data shows that, on average across participants, there were approximately 3.05 bleeding episodes per year during the main treatment phase. When looking only at bleeds that happened without an obvious cause (called spontaneous bleeds), the reported average was about 1.84 per year. For a smaller group of participants who received two or fewer doses per week, the reported average was around 4.1 bleeding episodes per year. The reported data also shows that the typical (median) time between doses was approximately 83 hours — roughly every three and a half days. The average weekly dose used was reported as 97.7 IU/kg (international units per kilogram of body weight, a standard way of measuring clotting factor doses). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02622321 · results posted 24 November 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 113 people in total across four groups. The trial involved people with a bleeding disorder called haemophilia A who also had inhibitors (meaning their body resists a standard treatment). Three groups (Arms A, C, and D) received a medicine called emicizumab given by injection once a week, while one group (Arm B) received no preventive treatment at first, then later switched to emicizumab. The trial was primarily measuring how often bleeds occurred over the course of a year — a figure called the annualised bleed rate, or ABR, which is simply an estimate of how many bleeds a person might experience in a 12-month period. The reported data shows that for the main comparison — Arm A (emicizumab) versus Arm B (no preventive treatment) — the estimated number of bleeds requiring treatment per year was 2.9 for the emicizumab group and 23.3 for the no-treatment group. When all bleeds (treated and untreated) were counted, the reported figures were 5.5 per year for the emicizumab group and 28.3 per year for the no-treatment group. For joint bleeds specifically requiring treatment, the reported figures were 0.8 per year (emicizumab) versus 6.7 per year (no treatment). The reported data also shows comparisons within the same participants before and after starting emicizumab: people in Arm A who had previously used on-demand treatment reported a drop from 37.7 all bleeds per year to 4.1, and treated bleeds dropped from 21.6 to 1.7. For Arm C participants who had previously used a different type of regular preventive treatment, all bleeds per year went from a reported 24.3 down to 5.5 on emicizumab. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01467427 · results posted 17 November 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 25 children with haemophilia B (a condition where the blood does not clot properly due to low levels of a protein called Factor IX). Twelve children were aged 0–6 years and 13 were aged 7–12 years. The trial was testing a treatment called nonacog beta pegol (also referred to as N9-GP), and it was primarily looking at whether children developed "inhibitors" — that is, antibodies (proteins made by the immune system) that could block the treatment from working. It also tracked bleeding episodes and how the body absorbed the treatment. The reported data shows that, for the primary outcome, none of the children in either age group (0 out of 12 younger children, and 0 out of 13 older children) developed these inhibitory antibodies during the trial. For the secondary outcomes, the reported average number of bleeding episodes per person per year was 0.33 for the younger group and 0.78 for the older group. When bleeding episodes were treated and rated on a four-point scale, the reported data shows that approximately 90% of bleeding episodes in younger children and approximately 90% in older children were rated as either "excellent" or "good" responses. The trial also measured how quickly and how much Factor IX entered the bloodstream after a dose; the reported figures for this absorption measure were very similar between the two age groups (0.015 and 0.016 in the relevant units). Lowest recorded Factor IX levels just before a dose was due were reported as 0.084 U/mL in the younger group and 0.109 U/mL in the older group. It is worth noting that of the 25 children who started the trial, only 10 completed the full study (including an extension phase), and the reasons for the others not completing were not detailed in the data provided here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01386528 · results posted 24 July 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 13 participants, all of whom completed the study. All 13 received a treatment called nonacog beta pegol, which is a clotting factor product. The trial was looking at how well bleeding was controlled during surgery in people who received this treatment, and also tracked things like how much of the treatment was used, whether blood transfusions were needed, and what unwanted health events occurred. The reported data shows that when surgeons and medical staff rated bleeding control right after surgery using a four-point scale (excellent, good, moderate, or poor), 10 out of 13 participants were rated "excellent" and the remaining 3 were rated "good." No participants were rated "moderate" or "poor." On average, participants received around 328 units of the treatment per kilogram of body weight across the whole surgical period. The reported data also shows that some participants did require blood transfusions — on average, around 275 mL during surgery and around 267 mL during the days following surgery. Average haemoglobin levels (a measure of red blood cells in the blood) were reported as 8.98 mmol/L before surgery, 8.37 mmol/L shortly after, and 7.99 mmol/L later in recovery. Regarding unwanted health events, the reported data shows a rate of approximately 12 adverse events (any unintended health issue noted during the trial) per patient year of exposure, while the rate of serious adverse events was reported as zero. It is important to note that this trial involved only 13 people, which is a very small group, and the figures above simply describe what was recorded and reported — they do not tell us what would happen in a broader population. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02020369 · results posted 14 June 2017

  According to the results reported on ClinicalTrials.gov, this trial involved 27 people in total (14 in one group and 13 in the other), with 22 completing the study. Participants were split into two groups, each receiving a different dose of a clotting treatment called FVIIa — either a lower dose (75 micrograms per kilogram of body weight) or a higher dose (225 micrograms per kilogram). The trial used a "crossover" design, meaning participants tried both doses at different times. The main thing being measured was how often mild or moderate bleeding episodes were considered successfully treated, based on a combination of the patient's own rating, whether extra treatment was needed, and whether bleeding appeared to have stopped. The reported data shows that, for the main measure, around 85 in every 100 bleeding episodes treated with the lower dose were rated as successfully treated, compared with around 93 in every 100 episodes treated with the higher dose. For a secondary measure — patients rating their own response as "good" or "excellent" at 12 hours — the reported figures were similar: about 86 in 100 for the lower dose and about 94 in 100 for the higher dose. The reported data also shows that participants on the higher dose reached a "good" or "excellent" response in a reported average of 3 hours, compared with about 6 hours for the lower dose. On average, the lower dose required about 2.5 doses per bleeding episode, while the higher dose required about 1.4 doses per episode. The total amount of drug used per bleeding episode was reported as approximately 188 micrograms per kilogram for the lower dose group and approximately 253 micrograms per kilogram for the higher dose group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01154231 · results posted 11 April 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 314 participants, all of whom received a treatment called BeneFIX (also known as Nonacog Alfa). Of those, 312 completed the study and 2 did not. The trial was measuring how often bleeding episodes occurred during a type of regular, scheduled treatment (called periodic replacement therapy), how many doses of the treatment were typically needed to stop a bleed, and how doctors rated the treatment's effect on bleeding episodes. The reported data shows that, on average, participants experienced 2.00 bleeding events per year while on the scheduled treatment programme. When a bleeding episode did occur, an average of 1.8 doses of BeneFIX were reported as being needed to bring the bleed under control. For the secondary outcome, the reported data shows that in 93.7% of cases, the treating doctor rated the effect of BeneFIX on a bleeding episode as either "excellent" or "good" — meaning roughly 9 out of every 10 treated bleeds received one of the two top ratings from the doctor involved. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01064284 · results posted 7 April 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 251 people with haemophilia A who had little or no prior exposure to clotting factor treatments. Of these, 125 received a plasma-derived von Willebrand factor/Factor VIII concentrate (pd vWF/FVIII — a treatment made from donated human blood) and 126 received a recombinant Factor VIII concentrate (rFVIII — a laboratory-manufactured version). The trial was primarily measuring how often participants developed "inhibitors" — antibodies that the body produces against the clotting factor treatment, which can reduce its usefulness. This was tracked over either 50 treatment sessions or three years, whichever came first. Around 107–109 participants in each group completed the trial. The reported data shows that in the pd vWF/FVIII group, 29 out of 125 participants developed inhibitors, compared with 47 out of 126 in the rFVIII group. Among those who did develop inhibitors, the reported data shows that 7 people in the pd vWF/FVIII group and 12 in the rFVIII group developed what are called "transient inhibitors" — meaning the inhibitors disappeared on their own within six months without additional treatment. In both groups, inhibitors were reported to appear at the same point on average — after 8 treatment sessions. The strength of the inhibitors at the time they were first detected was reported as 12 Bethesda Units (a standard measurement unit) in the pd vWF/FVIII group and 16.3 Bethesda Units in the rFVIII group. Two of the secondary outcomes — the anamnestic response (how the immune system reacted in inhibitor patients over time) and clinical factors linked to inhibitor development — had no data reported on ClinicalTrials.gov. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01439971 · results posted 6 April 2017

  According to the results reported on ClinicalTrials.gov, this trial tested a drug called PF-05280602, given as a single dose at five different dose levels (0.5, 4.5, 9.0, 18.0, and 30.0 micrograms per kilogram of body weight). A total of 29 people were enrolled across the five groups, with 25 going on to receive a dose and complete the trial. The trial was primarily measuring changes in basic body signs — blood pressure, body weight, body temperature, breathing rate, heart rate, and findings from physical examinations — to see how these measurements shifted after participants received the drug. The reported data shows that changes across all of these measurements were generally small across all five dose groups. For blood pressure, the reported changes from starting levels ranged from around −8.7 mmHg (millimetres of mercury, the standard unit for blood pressure) to small rises of a few mmHg, depending on the group and the specific measurement time point. Body weight changes were modest, ranging from about −0.4 kg to −1.0 kg across groups. Body temperature shifted by less than half a degree Celsius in any group. Breathing rate changed by between 0 and about 2 extra breaths per minute, and heart rate changes ranged from a small decrease of about 1.3 beats per minute to an increase of around 8.8 beats per minute. Physical examinations showed no reported changes from the previous check in any of the five groups. It should be noted that the submitted data included only partial time-point results for some measures, so not all figures across all time points were reported. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02492984 · results posted 4 April 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT02492984) enrolled 87 people with haemophilia A — 73 in an "on-demand" group (meaning they received treatment only when a bleed occurred) and 14 in a "surgical prophylaxis" group (meaning they received treatment around the time of surgery). The trial was measuring whether participants developed antibodies against the clotting treatment (called Factor VIII inhibitors), how participants responded to bleed treatments and surgery, and what adverse (unwanted) medical events occurred. Of the 87 who started, 84 completed the study. The reported data shows that, for the primary outcome — the proportion of participants who developed Factor VIII inhibitors (antibodies that can reduce how well the treatment works) — the figure was 8.22% in the on-demand group and 7.14% in the surgical prophylaxis group. For adverse events (any unwanted medical occurrence during the study), 65 out of 73 participants in the on-demand group and 10 out of 14 in the surgical prophylaxis group experienced at least one such event; serious adverse events were recorded in 7 and 1 participants respectively. Regarding bleed treatment response in the on-demand group, 46.9% of infusions (doses) were rated "excellent," 40.0% "good," 12.7% "moderate," and 0.3% "no response," with 0.1% not categorised. On average, 1.6 infusions were needed per bleed, and 63% of bleeds were resolved with just one infusion. For the surgical group, 71.4% of intraoperative (during surgery) assessments were rated "excellent" and 28.6% "good," with similar figures reported post-operatively (75.5% excellent, 24.5% good). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02093897 · results posted 27 January 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 84 adult participants, all of whom received a clotting factor treatment called rVIII-SingleChain, which is a type of Factor VIII (a protein the blood needs to clot properly) used to manage haemophilia A. Of the 84 who started, 65 completed the study and 19 did not. The trial was measuring how well the treatment controlled bleeding episodes, both when used as a regular preventive (prophylaxis) regimen and when used on-demand (only when a bleed occurred). The reported data shows that, across 347 treated bleeding episodes in the group assessed for effectiveness, 96.3% were rated as a treatment success — meaning the investigator judged the outcome as either "excellent" or "good" on a four-point scale. Looking at how many infusions (doses) were needed per bleeding episode, the reported data shows that 85.9% of bleeds were managed with just one infusion, 9.8% needed two, 2.3% needed three, and 2.0% required more than three. For the annualised bleeding rate — that is, the estimated number of treated bleeds per year — the reported data shows 78.56 bleeds per year in the on-demand group and 3.69 per year in the prophylaxis group. The reported data also shows that participants on the on-demand regimen used an average of approximately 2,429 IU/kg (units of the treatment per kilogram of body weight) per year, while those on prophylaxis used approximately 4,541 IU/kg per year. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01405742 · results posted 19 September 2016

  According to the results reported on ClinicalTrials.gov, this trial enrolled 4 people in total — 2 in each group. The study was looking at two different dosing schedules for a clotting factor treatment (called Factor VIII, or FVIII) used in haemophilia: taking it once a week versus three times a week. Participants crossed over between the two schedules during the trial. Three out of four participants completed the study; one person in the once-weekly-first group did not finish. The reported data shows that the main thing being measured was how many bleeds occurred over a 26-week period. The once-weekly group recorded 3 bleeds in total across all participants in that phase, while the three-times-weekly group recorded 16 bleeds in total across that phase. It is important to note that these are combined totals across all participants, not figures per individual. Two secondary measures were also reported. The first looked at something called thrombin generation — a lab test that measures the blood's clotting activity — with a reading of 110.65 nMs for the once-weekly phase and 82.86 nMs for the three-times-weekly phase. The second secondary measure was the level of Factor VIII activity in the blood, recorded as 1.04 IU/mL for the once-weekly phase and 0.90 IU/mL for the three-times-weekly phase. Because only 4 people took part in this trial, the numbers above represent a very small group, and the reported data should be understood in that context. The trial appears to have been an early-stage study focused on gathering initial measurements rather than drawing broad conclusions. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01736475 · results posted 7 September 2016

  According to the results reported on ClinicalTrials.gov, this trial (NCT01736475) enrolled 138 people in total — 121 in a "prophylaxis" group (receiving regular scheduled infusions of a treatment called BAX 855) and 17 in an "on-demand" group (receiving infusions only when a bleed occurred). The trial's main goal was to measure the annualised bleeding rate (ABR) — that is, roughly how many bleeding episodes each person experienced per year, on average — and to compare that figure between the two groups. The reported data shows that participants in the prophylaxis group had an average of 4.3 bleeds per year, while those in the on-demand group had an average of 43.4 bleeds per year. For a secondary measure — looking at how well BAX 855 controlled individual bleeding episodes — the reported data shows that 96% of bleeding episodes assessed were rated as having an "excellent" or "good" response 24 hours after treatment began (meaning full or clear improvement in pain and signs of bleeding was recorded). On average, participants in the prophylaxis group needed about 1.37 infusions to treat a bleeding episode, and those in the on-demand group needed about 1.21 infusions per episode. The average dose used per prophylactic infusion was reported as approximately 44.51 IU per kilogram of body weight. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01488994 · results posted 1 September 2016

  According to the results reported on ClinicalTrials.gov, this trial involved 23 children with haemophilia B who received a treatment called BAX326, a clotting factor product. The children were split into two age groups: 11 children under 6 years old, and 12 children aged 6 to under 12 years old. All 11 of the younger children completed the study, while 11 of the 12 older children completed it (one did not finish). The trial was measuring how the body processed BAX326 and recording any side effects considered possibly or probably linked to the treatment. The reported data shows that the primary outcome — the number of side effects judged to be possibly or probably related to BAX326 — was zero across both age groups and the overall group combined. For the secondary outcomes, the trial tracked how the body handled the medication over time. The reported data shows that in children under 6, the drug remained active in the bloodstream for an average of about 30.6 hours, compared to about 25.3 hours in the older group (combined average: about 27.9 hours). The rate at which the body cleared the drug was reported as 0.1058 units per kilogram per hour in the younger group and 0.0874 in the older group. One secondary measure — how much the drug's activity levels rose per unit of dose given — was reported as 0.586 in the younger group and 0.731 in the older group. One pharmacokinetic measure (AUC 0–72 hours per dose) had no data reported for any group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02083965 · results posted 19 August 2016

  According to the results reported on ClinicalTrials.gov, this trial enrolled 19 people in total — 10 in one group and 9 in the other. The trial was looking at how the body processed two different doses of a clotting factor treatment called rFVIIIFc (1,000 IU and 3,000 IU). This type of study is called a pharmacokinetic (PK) study, meaning it tracked how the treatment moved through the body over time — for example, how quickly it was absorbed, how long it stayed in the bloodstream, and how quickly the body cleared it. All 19 participants completed the measurement phases of the trial, and 17 of the 18 who entered the final treatment period also completed it. The reported data shows the following figures for the two doses. The total exposure to the treatment over time (a measure called "area under the curve") was reported as 2,888.9 units for the 1,000 IU dose and 2,646.3 units for the 3,000 IU dose. The peak level of the treatment detected in the blood was 122.2 IU/dL for the lower dose and 129.08 IU/dL for the higher dose. The reported "half-life" — meaning the time it took for the level in the blood to drop by half — was around 18.3 hours for the 1,000 IU dose and 17.5 hours for the 3,000 IU dose. How quickly the body cleared the treatment was reported as 1.807 mL/h/kg and 2.016 mL/h/kg respectively. The measure of how the treatment spread through the body (volume of distribution) was reported as 47.00 mL/kg and 48.65 mL/kg for the two doses. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01486927 · results posted 9 August 2016

  According to the results reported on ClinicalTrials.gov, this trial (NCT01486927) enrolled 174 people with haemophilia A (a condition where the blood does not clot properly) who received a recombinant Factor VIII product called rVIII-SingleChain. Of those who started, 161 completed the trial and 13 did not. The trial was measuring several things: how well bleeding episodes were controlled, whether participants developed antibodies (called inhibitors) against the treatment, how often spontaneous bleeds occurred per year, and how the treatment performed during surgery. A smaller group also had their blood tested to see how the treatment moved through the body over time. The reported data shows that across all participants, around 92% of bleeding episodes were rated by the investigator as "excellent" or "good" in terms of how they responded — this rating applied whether participants were on a regular dosing schedule (prophylaxis) or were only treating bleeds as they occurred (on-demand). For the on-demand group, the reported average number of spontaneous bleeds per year was 11.73, while for those on the regular dosing schedule it was reported as 0.00. In the surgical sub-study, which covered 16 operations, 100% of surgeries received an "excellent" or "good" rating. Importantly, the reported data shows that none of the 174 participants developed inhibitors (antibodies that can reduce how well Factor VIII works) during the trial period. The reported data also includes some blood-level measurements from a pharmacokinetic (how the body processes a medicine) sub-study, showing figures for how much of the treatment was present in the blood over time and the peak level reached, though the full details of these comparisons were not broken down further in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02213250 · results posted 25 July 2016

  According to the results reported on ClinicalTrials.gov, this trial (NCT02213250) looked at how the body processes a medicine called BeneFIX (a clotting factor treatment given by injection) in two age groups of participants. A total of 12 people took part — 4 children aged 6 to under 12 years, and 8 people aged 12 years and older. All 12 participants completed the study. The trial was measuring what is known as "pharmacokinetics" — that is, how the medicine moves through the body over time after a single dose of 50 IU per kilogram of body weight, including how high the levels in the blood rose and how long the medicine stayed in the body. The reported data shows the following measurements for the two age groups. The peak level of the medicine in the blood (the highest concentration reached) was 0.388 IU/mL in the younger group and 0.423 IU/mL in the older group. The total exposure to the medicine over time — measured in two ways — was lower in the younger group (6.93 and 7.84 IU·hr/mL) compared to the older group (9.71 and 11.66 IU·hr/mL). The time it took to reach that peak level was 0.5 hours in the younger group and 0.375 hours in the older group. The estimated "volume of distribution" — a way of describing how widely the medicine spread through the body relative to body weight — was 227.9 mL/kg in the younger group and 218.8 mL/kg in the older group. The rate at which the medicine left the body was also slightly different between the groups (0.025 per hour in the younger group versus 0.018 per hour in the older group), suggesting the medicine cleared somewhat more slowly in the older participants. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01662531 · results posted 9 May 2016

  According to the results reported on ClinicalTrials.gov, this trial (NCT01662531) enrolled 27 children with haemophilia B (a condition where the blood lacks enough of a clotting protein called Factor IX). All 27 participants completed the study — none dropped out. The trial was investigating how the body processed a medicine called rIX-FP, a laboratory-made form of Factor IX. Researchers looked at things like how quickly the medicine entered the bloodstream, how long it stayed active in the body, and whether the children's immune systems produced substances (called inhibitors) that could block the medicine from working. The reported data shows that after a single dose of rIX-FP, the medicine remained active in the blood for an average of around 91 hours (roughly 3.8 days), compared to approximately 19 hours for the children's previous Factor IX product. The reported incremental recovery — a measure of how much the clotting protein level in the blood rose per unit of medicine given — was approximately 1.01 for rIX-FP overall, compared to about 0.74 for the previous product. These figures were broadly similar across the two age groups tested (children under 6, and children aged 6 to under 12). The reported data also shows that zero out of 27 participants developed inhibitors to Factor IX during the study. Regarding side effects, the data reports that 26 out of 27 participants experienced at least one adverse event (an unwanted health event recorded during the trial); however, zero participants experienced an adverse event that was considered treatment-related, according to the reported results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01410227 · results posted 21 April 2016

  According to the results reported on ClinicalTrials.gov, this trial (NCT01410227) enrolled 37 people across four groups, testing a recombinant (laboratory-made) form of a blood-clotting protein called von Willebrand Factor (rVWF), given either alone or combined with another clotting protein called Factor VIII (rFVIII). The four groups received different combinations and dose levels of these study products. Of the 37 who started, 30 completed the study — 4 out of 8 in the first group, all 8 in the second, 13 out of 15 in the third, and 5 out of 6 in the fourth. The reported data shows that the main thing being measured was whether bleeding episodes were brought under control, using a scoring system based on how many infusions (drip treatments) were needed compared to what was expected. According to the results reported on ClinicalTrials.gov, 100% of participants in the analysed group met the pre-defined threshold for "treatment success" on this scoring system. For the secondary measures, the reported data shows that 100% of treated bleeding episodes — both overall and when gut bleeds were excluded — were rated "excellent" or "good" on the same scale. On average, each bleeding episode required 1 infusion of the study product, at a reported average dose of 48.2 units per kilogram of body weight. Regarding a safety-related measure — whether participants developed antibodies (proteins the body can produce that may interfere with a treatment) against Factor VIII — the reported data shows 0% of participants tested positive. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02460458 · results posted 19 April 2016

  According to the results reported on ClinicalTrials.gov, this trial (NCT02460458) involved people diagnosed with Type 3 Von Willebrand's Disease (VWD3) — a rare inherited condition where the blood is missing or very low in a protein called Von Willebrand Factor, which is needed for normal clotting. The trial ran in several phases: it started with 265 participants, and the number who completed each successive phase reduced over time, with 92 participants completing the final phase. The trial was not testing a new treatment; instead, it was focused on measuring specific proteins in participants' blood to better understand and confirm the diagnosis of this condition. The reported data shows the average levels of several blood proteins measured across participants. The Von Willebrand Factor protein level (VWF:Ag) had a reported average of 1.01 IU/dL (IU/dL is a unit used to measure how much of a substance is in the blood), and the Von Willebrand Factor Propeptide — a related molecule that helps characterise the condition — averaged 6.44 IU/dL. Two measures of a related clotting protein called Factor VIII were also reported: one test showed an average of 2.42 IU/dL, another method gave 1.54 IU/dL, and a third measure of the Factor VIII protein itself averaged 3.63 IU/dL. All of these measurements were only taken in participants who already had levels at or below 5 IU/dL, as part of the diagnostic criteria. The trial also looked at the physical structure of the Von Willebrand Factor protein — 174 participants showed one pattern, 54 showed another, and 15 showed a third. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00994929 · results posted 2 March 2016

  According to the results reported on ClinicalTrials.gov, this trial involved 9 participants, all of whom completed the study with no drop-outs. The trial was looking at a drug called Neumega (also known as Oprelvekin or Interleukin 11) and measuring its biological effects in the blood — specifically focusing on a protein called von Willebrand factor (VWF), which plays a role in how blood clots. The study also tracked any unwanted side effects and looked at changes in the genetic activity related to this protein. The reported data shows that two measurements were taken for the main outcome (VWF activity in the blood), reported as a percentage compared to normal healthy blood levels (where 100% equals normal). The two figures reported were 34.75% and 143.4%, though the data as submitted does not clearly distinguish which measurement these two figures represent separately (for example, before and after treatment). For the secondary outcomes, the reported data shows that 6 out of 9 participants experienced at least one adverse event (an unwanted or unexpected health occurrence noted during the trial). The study also measured changes in genetic activity related to VWF and reported a figure of 0.81-fold increase, meaning the level of genetic activity measured was slightly below what would be considered a doubling. No further breakdown of these figures was provided in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02165462 · results posted 15 December 2015

  According to the results reported on ClinicalTrials.gov, this trial enrolled 17 people with haemophilia, and all 17 completed the study — meaning no one dropped out. The trial was measuring how force is produced through the legs during movement, using a specialised pressure platform, as well as the condition of joints using a standardised scoring tool called the Haemophilia Joint Health Score (HJHS). The HJHS runs from 0 to 24, where 0 means no joint damage and 24 means the most severe joint damage possible. The reported data shows that when it came to leg force measurements, the group recorded an average maximal peak force of 15.67 Newtons per kilogram of body weight. The trial also measured how evenly force was shared between the left and right legs — a figure called the "bilateral index." The reported data shows a bilateral index of maximal peak force of −40.86%, and bilateral indexes for the rate of force build-up of 31.34% (during a preparation phase) and 20.05% (during an acceleration phase). A figure further from zero in these bilateral index measurements suggests greater imbalance between the two sides of the body. For the joint health scores, the reported data shows four separate measurements recorded across the group, with values of 4.88, 3.00, 5.82, and 6.35 on the 0–24 scale, though the data as submitted does not specify which joints or time points these figures individually correspond to. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01440946 · results posted 18 June 2015

  According to the results reported on ClinicalTrials.gov, this trial enrolled 30 children with haemophilia B (a condition where the blood does not clot properly due to low levels of a protein called Factor IX). There were two age groups: 15 children under 6 years old, and 15 children aged 6 to under 12 years old. The trial was measuring whether a treatment called rFIXFc caused the immune system to develop "inhibitors" — antibodies that can block the treatment from working — and also tracked how often bleeding episodes occurred and how much of the treatment was used. Of the 30 children who started, 13 in the younger group and 14 in the older group completed the study. The reported data shows that the primary thing being measured — whether any child developed a confirmed inhibitor — was reported as 0% in both age groups, meaning no confirmed inhibitors were recorded in either group. For bleeding episodes, the reported average number of bleeds per child per year was 1.09 in the younger group and 2.13 in the older group. When looking only at spontaneous (unprompted) joint bleeds, the reported average was 0.00 per year in both groups. In terms of how caregivers rated the response to the first injection given to treat a bleed, around 89.5% of first injections in the younger group and 88.2% in the older group were rated as having some level of response. Doctors' overall assessments rated the treatment regimen as "excellent" for approximately 85% of responses in the younger group and 90% in the older group. The reported data also shows the average annual amount of the treatment used: roughly 3,042 international units per kilogram (a standard measure of dosing) in the younger group and 3,186 in the older group for routine prevention of bleeds, with smaller additional amounts used for treating bleeds when they occurred. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01335061 · results posted 4 May 2015

  According to the results reported on ClinicalTrials.gov, this trial (NCT01335061) enrolled 25 participants, all of whom completed the study — none dropped out. The trial was looking at a clotting factor medicine called BeneFIX (nonacog alfa, recombinant factor IX), which is used by people with haemophilia B (a condition where the blood does not clot properly). The study compared two ways of using the medicine: "on-demand" treatment, meaning the medicine was only given after a bleed started, versus "prophylaxis" treatment, meaning the medicine was given on a regular schedule to try to prevent bleeds before they happened. The reported data shows that the average number of bleeding episodes per year was 32.9 during the on-demand period and 3.6 during the prophylaxis period. For the on-demand treatment, responses to individual bleeding episodes were rated on a four-point scale (Excellent, Good, Moderate, or No response). The reported data shows that for first infusions treating a bleed, there were 271 observations rated Excellent, 177 rated Good, 55 rated Moderate, 3 rated as No response, and 1 observation with data not clearly categorised. For follow-up infusions, the corresponding numbers were 39, 80, 33, 0, and 0. The reported data also shows that most bleeds requiring on-demand treatment were handled with a single infusion (416 bleeds), while smaller numbers needed 2, 3, 4, or more than 4 infusions (69, 9, 3, and 10 bleeds respectively). Only 1 breakthrough bleed (an unexpected bleed occurring shortly after a scheduled prophylaxis dose) was reported within 48 hours of a prophylaxis dose. The reported data shows that the average dose per infusion was 52 IU/kg during on-demand treatment and 99 IU/kg during prophylaxis treatment. The total annualised amount of medicine used per person was reported as 707 IU/kg per year during on-demand treatment and 4,985 IU/kg per year during prophylaxis treatment. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01810666 · results posted 5 January 2015

  According to the results reported on ClinicalTrials.gov, this trial (NCT01810666) enrolled 30 participants, and all 30 completed the study. The trial looked at a recombinant (laboratory-made) Factor VIII product — a clotting protein used in haemophilia A — and compared two approaches: treating bleeds only when they happened ("on-demand") versus taking the treatment regularly to try to prevent bleeds ("prophylaxis"). Each participant went through both approaches in sequence, so the study could compare the same people under both conditions. The reported data shows that, on average, participants had 56 more bleeds per year during the on-demand period than during the prophylaxis period. When looking only at bleeds into joints (such as knees, elbows, or ankles), the reported difference was about 37.71 fewer joint bleeds per year during prophylaxis compared with on-demand treatment. The trial also measured joint health using a scoring tool called the Hemophilia Joint Health Score, which runs from 0 to 124, where higher numbers indicate poorer joint condition. The reported data shows a median difference of −3.00 points in how joint scores changed between the two periods, meaning scores moved in a slightly more favourable direction during prophylaxis, though the full confidence interval (the range within which the true figure likely sits) was not reported in the data provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00486278 · results posted 25 December 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 51 people, of whom 42 completed the study and 9 did not. The trial was looking at different doses of an investigational medicine called vatreptacog alfa (tested at five dose levels: 5, 10, 20, 40, and 80 micrograms per kilogram of body weight) and comparing it to an existing medicine called rFVIIa (at 90 micrograms per kilogram). Both medicines are related to blood clotting. The trial's primary focus was counting the number of unwanted medical events (called adverse events) that occurred across the groups, and secondary measurements looked at various blood clotting markers as well as how many doses of medicine were needed to bring a bleed under control. The reported data shows that the number of adverse events varied across groups: the 5 mcg/kg vatreptacog alfa group recorded 10 events, the 10 mcg/kg group recorded 8, the 20 mcg/kg group recorded 5, the 40 mcg/kg group recorded 5, the 80 mcg/kg group recorded 0, and the rFVIIa comparison group recorded 11. For the blood clotting marker measurements, the data shows that levels of the investigational medicine in the blood and changes in clotting time tests (such as prothrombin time and partial thromboplastin time) varied across dose groups before and after dosing. For example, a clotting speed measurement (prothrombin time, expressed as a percentage where higher numbers suggest faster clotting activity) rose from around 80% before dosing to around 128% after dosing in the highest vatreptacog alfa dose group, and to 139% in the rFVIIa comparison group. For bleeding episodes, the number of doses needed to stop a bleed was also reported across groups, though the total numbers of episodes assessed were small in each group, ranging from 2 to 9 episodes per group. It is worth noting that some secondary outcome measurements were not reported for all dose groups in the data submitted to ClinicalTrials.gov — for instance, blood activity level data was only provided for the 20 mcg/kg dose and above. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01458106 · results posted 12 December 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 71 boys with haemophilia A — 36 children under 6 years old and 35 children aged 6 to under 12 years old. The trial was measuring how a clotting factor treatment called rFVIIIFc performed in these two age groups, with the main focus on whether children developed "inhibitors" — antibodies that can block the treatment from working. Most children completed the study (33 in the younger group and 34 in the older group). The reported data shows that, for the primary outcome, zero per cent of participants in either age group developed confirmed inhibitors, both among those who received 50 or more doses and among all participants overall. For bleeding episodes, the reported annualised bleeding rate (the estimated number of bleeds per person per year) was 0.00 for children under 6 and 2.01 for children aged 6 to under 12. Spontaneous joint bleeds were reported as 0.00 per person per year in both groups. When caregivers rated how well the first injection controlled a bleed, around 91% of responses for the younger group and 94% for the older group were rated as either "excellent" or "good." Doctors' assessments of the overall treatment regimen were rated "excellent" for approximately 97% of responses in the younger group and 90% in the older group. The reported annual amount of the treatment used was approximately 5,332 units per kilogram per year for the younger group and 4,974 units per kilogram per year for the older group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01027364 · results posted 4 September 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 123 participants across four treatment groups. Sixty-three people were in the weekly preventive dosing group (Arm 1), 29 in the personalised-interval preventive dosing group (Arm 2), 27 in the on-demand (treat-when-bleeding) group (Arm 3), and 4 in a surgery management group (Arm 4). The trial was measuring things like abnormal blood test results and any unwanted medical events that occurred during the study, as well as how well bleeding episodes appeared to respond to injections of the study medicine (rFIXFc), a clotting factor treatment for haemophilia B. The reported data shows that, for potentially concerning laboratory (blood test) abnormalities, small numbers of participants were affected across the main three arms — for example, 2 participants in Arm 1, none in Arm 2, and 2 in Arm 3 showed one type of abnormality, while other specific abnormalities were seen in 1, 2, or 3 participants respectively. For unwanted medical events (called adverse events) that emerged during the study, 45 participants in the rFIXFc part of Arm 1, 23 in Arm 2, 20 in Arm 3, and 10 in Arm 4 reported at least one such event. Serious adverse events were reported in 5 participants in the rFIXFc part of Arm 1, 4 in Arm 2, 1 in Arm 3, and none in Arm 4. For the surgery group specifically, 2 participants had non-serious events and 1 had a serious event during the surgical period. For the secondary outcomes, the reported data shows that when participants rated how their bleeding episodes responded to the first injection, the percentage rated as "excellent or good" combined was around 78.8% in Arm 1, 74.6% in Arm 2, and 87.1% in Arm 3. When doctors rated each participant's overall response to treatment at the end of the study, approximately 74.5% of assessments in Arm 1, 73.2% in Arm 2, and 58.3% in Arm 3 were rated "excellent," with no assessments rated "ineffective" across any of the three arms. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00884390 · results posted 1 September 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 208 people with haemophilia A (a condition where the blood does not clot properly). Of these, 146 were in the "ReFacto Switch" group — people switching from an older version of a clotting medicine to a newer formulation called moroctocog alfa — and 62 were in the "Other Switch" group, meaning people switching from a different clotting medicine to the same newer formulation. The trial's main question was whether switching to the newer formulation caused the body to develop "inhibitors" — antibodies that can block the medicine from working. By the end, 123 people in the ReFacto Switch group and 54 in the Other Switch group completed the study. The reported data shows that, for the primary outcome, zero participants in either group developed what the trial defined as a clinically significant inhibitor during the study period. For the secondary outcomes, the annualised bleeding rate — that is, the estimated number of bleeds per year — was reported as 28.35, 1.08, and 8.43 across different categories, though the data as submitted does not fully label which figure belongs to which subgroup. Among participants who received at least one preventive (prophylactic) dose, 33 people experienced a "breakthrough bleed" (a bleed that occurred despite preventive treatment), and 96 such bleeding episodes were recorded within 48 hours of a preventive infusion. When bleeds were treated on demand, the reported data shows the first infusion response was rated as excellent for 1,031 observations, good for 1,650, moderate for 191, and no response for 26, out of 3,241 total observations recorded. It is worth noting that some of the secondary outcome figures lack complete labelling in the submitted data, so a full breakdown cannot be provided here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01181128 · results posted 29 August 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 165 adult men with severe haemophilia A (a condition where the blood does not clot properly). Participants were split into three groups: 118 people received a personalised (tailored) preventive dosing schedule, 24 received a fixed weekly preventive dose, and 23 received treatment only when a bleed occurred (on-demand). The trial was measuring how often bleeds happened, whether participants developed antibodies (called "inhibitors") that could interfere with the treatment, and a range of safety monitoring measures including side effects and changes in blood test results and vital signs. The reported data shows that across all three groups, zero participants developed inhibitory antibodies against the clotting factor treatment, whether measured among those with 50 or more days of exposure or across all participants regardless of exposure. For bleeding episodes, the reported average number of bleeds per person per year was approximately 1.6 in the tailored preventive group, 3.6 in the weekly preventive group, and 33.6 in the on-demand (treat when bleeding) group. Regarding side effects, the reported data shows that 80 participants in the tailored preventive group experienced at least one treatment-emergent adverse event (an unwanted medical occurrence that appeared or worsened after starting the study treatment), as did 18 in the weekly group and 10 in the on-demand group. Serious adverse events were reported in 5, 3, and 2 participants in those respective groups. Small numbers of participants across the groups also had notable changes in laboratory blood test results or vital signs such as blood pressure and pulse, with the specific figures varying by group and measurement type. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01341912 · results posted 3 February 2014

  According to the results reported on ClinicalTrials.gov, this trial involved 3 participants, all of whom completed the study with no drop-outs. The trial was looking at a treatment called Human-cl rhFVIII, which is a type of clotting factor replacement. The study was measuring two main things over the long term: whether participants developed "inhibitors" (antibodies that can block the treatment from working) and how well the treatment appeared to respond to bleeding episodes. The reported data shows that when it came to inhibitor development — the primary thing being measured — none of the 3 participants developed inhibitors during the study period. For the secondary measure, bleeding episodes were rated on a four-point scale (Excellent, Good, Moderate, or None). According to the results reported on ClinicalTrials.gov, approximately 71.9% of bleeding episodes were rated "Excellent," about 26.6% were rated "Good," around 1.6% were rated "Moderate," and 0% were rated "None." It is worth noting that with only 3 participants, these percentages are based on a very small number of people and bleeding events. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00989196 · results posted 17 January 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 22 people in total — 10 in one group and 12 in the other. The trial was comparing two clotting factor VIII (FVIII) products: an investigational product called Human-cl rhFVIII and an existing product called Kogenate FS. Both are treatments for haemophilia A, a condition where the blood does not clot properly. Each participant received both products at different times (a "crossover" design), so the trial could directly compare how each product behaved in the body. All 22 participants completed the initial comparison phase; one person from the first group did not complete the later treatment phase. The reported data shows that the main thing being measured was how much of each product circulated in the blood over time after a single infusion — a calculation known as "area under the curve" (essentially a way of adding up the total exposure to the product across many time points). For Human-cl rhFVIII this figure was reported as 0.39, and for Kogenate FS it was 0.38 (both in the same units). The trial also measured several other properties of how each product moved through the body. The reported half-life — meaning the time it takes for the amount in the blood to drop by half — was approximately 14.7 hours for Human-cl rhFVIII and 16.1 hours for Kogenate FS. The peak level reached in the blood was 1.462 IU/mL for Human-cl rhFVIII and 1.394 IU/mL for Kogenate FS, and both products reached that peak at roughly the same time (about 0.35 hours). The average time the product spent in the body was around 19.5 hours for Human-cl rhFVIII and 20 hours for Kogenate FS, and the estimated spread of the product through the body was 49.58 mL/kg versus 53.32 mL/kg respectively. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01392547 · results posted 6 December 2013

  According to the results reported on ClinicalTrials.gov, this trial enrolled 72 participants in total, with 64 completing the study and 8 not completing it. All participants received both treatments at different times — an experimental clotting agent called vatreptacog alfa (at a dose of 80 µg/kg) and an existing clotting agent called rFVIIa (at a dose of 90 µg/kg). The trial was measuring how well each treatment controlled bleeding episodes in people with a bleeding disorder, including whether bleeding stopped without needing any extra clotting medication, and how many doses were needed per bleed. The reported data shows that for the main measure — bleeding episodes that were controlled without any additional clotting medication — vatreptacog alfa was assessed across 318 bleeding episodes, of which 22 met that measure, while rFVIIa was assessed across 211 bleeding episodes, of which 16 met that measure. For the secondary measure of bleeding episodes that were controlled and stayed controlled, the reported data shows 53 out of 268 episodes for vatreptacog alfa, and 51 out of 163 episodes for rFVIIa. Regarding how many doses were needed per bleed, the data was reported in categories but the specific category labels were not included in the submitted data, so a full breakdown cannot be described here. The reported data also shows that 55 adverse events (unexpected medical occurrences during the trial) were recorded among participants receiving vatreptacog alfa, compared with 11 for rFVIIa. On the question of whether participants developed antibodies against the trial products — which can sometimes interfere with how a treatment works — 8 participants receiving vatreptacog alfa were reported to have developed antibodies against it, while no participants receiving rFVIIa were reported to have done so; one participant receiving rFVIIa was reported to have developed antibodies against vatreptacog alfa. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01029340 · results posted 18 November 2013

  According to the results reported on ClinicalTrials.gov, this trial tested a clotting protein medicine called BAY81-8973 (a recombinant Factor VIII, a laboratory-made version of a protein the body needs to help blood clot) in people with haemophilia A. The trial ran in several parts. Part A involved 28 people split into two groups to compare how the body processes BAY81-8973 versus an existing medicine called Kogenate FS. Part B involved 63 people across two groups, looking at bleeding episodes over time. A smaller Part C involved 5 people, and an extension period included 55 people. The trial was measuring things like how the drug moved through the body and how often participants experienced bleeds. The reported data shows that in Part A, when looking at how much of the drug was present in the blood over time (a measure called "area under the curve"), the figure for BAY81-8973 was 1,889 IU·h/dL, compared with 1,584 IU·h/dL for Kogenate FS. The reported half-life — meaning roughly how long it took for the amount of drug in the blood to reduce by half — was about 13.8 hours for BAY81-8973 and 12.0 hours for Kogenate FS. For Part B, the reported average annualised number of bleeds (that is, the estimated number of bleeds per person per year) was 1.03. When broken down by the two different dosing methods used in Part B, the annualised bleed count was reported as 1.9 in both groups. The reported data also shows that on average, participants needed 1.0 injection to treat a bleed, in both dosing groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00484185 · results posted 12 August 2013

  According to the results reported on ClinicalTrials.gov, this trial (NCT00484185) involved a single group of 183 participants who were enrolled, of whom 178 received the study drug BeneFIX (a recombinant Factor IX product used in people with haemophilia B). The trial was primarily measuring adverse events (that is, any unwanted medical occurrences that happened while participants were taking the study drug) across a range of participant characteristics, including age, sex, severity of bleeding, and medical history. A total of 112 participants completed the study, while 71 did not complete it. The reported data shows that when adverse events were grouped by how severe they were, 6 participants experienced mild events, 2 experienced moderate events, and 4 experienced severe events. When looking at how serious the events were — using a stricter definition that includes things like hospitalisation or life-threatening situations — 4 participants had serious adverse events and 5 had non-serious adverse events. Regarding the relationship between the adverse events and the study drug, the reported data shows 9 participants had adverse events considered unrelated to the drug, and 1 participant had an adverse event considered possibly related to the drug. The data for some outcome measures, including the specific actions taken in response to adverse events and the final outcomes of those events, was not reported in the structured results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00623480 · results posted 7 August 2013

  According to the results reported on ClinicalTrials.gov, this trial involved 84 people in total — 42 in each of two groups. All participants had haemophilia and were given a recombinant (laboratory-made) clotting protein called Factor VIII. One group received it on a regular preventive schedule (called prophylaxis), while the other group only received it when a bleed occurred (called on-demand treatment). The trial ran for three years and tracked how often bleeds happened, as well as changes in joint health and quality of life. Of the 84 who started, 70 completed the trial — 35 from each group. The reported data shows that, over the study period, the median number of bleeds (the middle value when all results are lined up) was 0 for the prophylaxis group and 54.5 for the on-demand group. For joint health, two different scoring tools were used — higher scores on both indicate more joint damage. On the MRI-based scale (ranging from 0 to 45), the reported average change from the start of the trial to three years was +0.79 in the prophylaxis group and +0.96 in the on-demand group. On the physical examination-based joint scale (ranging from 0 to 142), the reported average change was −0.31 in the prophylaxis group (a very small improvement) and +0.63 in the on-demand group (a small worsening). For the quality-of-life physical functioning measure (where higher scores mean better quality of life, on a 0–100 scale), the reported average change was +7.86 in the prophylaxis group and −5.30 in the on-demand group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00666406 · results posted 26 March 2013

  According to the results reported on ClinicalTrials.gov, this trial enrolled 9 participants in total — 6 assigned to receive Advate (rAHF-PFM) first and then Recombinate (rAHF), and 3 assigned to receive Recombinate first and then Advate. All 9 participants completed both phases of the study. The trial was measuring how two different recombinant (laboratory-made) clotting factor VIII products — used in the management of haemophilia A — behaved in the bloodstream over time after a single infusion of each product. Specifically, it tracked how much of the clotting factor was present in the blood over a 48-hour period, using several different laboratory testing methods. The reported data shows the main measurement used was the "area under the curve" (AUC) — a way of capturing the total amount of clotting factor circulating in the blood over time, expressed in units of IU\*h/dL. Using a standard central laboratory test (aPTT-based assay), the reported AUC over 48 hours was 1,104 IU\*h/dL for Advate and 1,294 IU\*h/dL for Recombinate. Two additional local laboratory tests gave figures of 1,180 vs 1,358 IU\*h/dL (chromogenic method) and 1,505 vs 1,664 IU\*h/dL (another clotting assay), while a fourth local method recorded 833 vs 901 IU\*h/dL. For the secondary outcomes — which extended the AUC calculation beyond 48 hours out to a theoretical endpoint — the reported figures were 1,190 vs 1,393 IU\*h/dL (central lab) and 1,282 vs 1,452 IU\*h/dL (local chromogenic method). It is worth noting that this was a very small study of only 9 people, so the numbers reflect a limited group. No conclusions about which product performed better or worse should be drawn from this plain summary alone. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00765726 · results posted 20 January 2012

  According to the results reported on ClinicalTrials.gov, this trial (NCT00765726) enrolled 12 participants, all of whom received a clotting factor treatment called Xyntha (a type of Factor VIII replacement). The trial was measuring whether participants developed "inhibitors" — which, in plain terms, means antibodies that the body can produce against the treatment, potentially making it less able to do its job. Of the 12 people who started, only 3 completed the trial, and 9 did not complete it. The data does not report the reasons why participants did not complete the study. The reported data shows that, among those assessed, 0% of participants developed these inhibitors during the trial period, based on a specific laboratory test used to detect them. It is worth noting that because only 3 of the 12 participants completed the trial, this result is based on a very small group of people, and no secondary outcome measure data appears to have been reported on ClinicalTrials.gov for this study. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00623727 · results posted 17 August 2011

  According to the results reported on ClinicalTrials.gov, this trial enrolled 70 people in one group and 73 in another — a total of 143 participants — all of whom had haemophilia A (a condition where the blood does not clot properly). The trial compared two versions of a clotting factor treatment given regularly to try to prevent bleeding: one version (BAY79-4980) used a special fat-based coating called pegylated liposomes, and the other (BAY14-2222) was a standard version mixed with plain water. The main thing the trial was measuring was the proportion of participants who had fewer than 9 total bleeds over the course of a year. The reported data shows that, for the primary measure, 38.1% of participants in the liposome-coated group had fewer than 9 total bleeds per year, compared with 72.1% in the standard group. For one of the secondary measures — the proportion of people who had fewer than 5 joint bleeds (bleeds into joints) per year — the reported figures were 38.1% for the liposome-coated group and 63.2% for the standard group. Among those who did meet the "fewer than 9 total bleeds" threshold, the reported average number of joint bleeds per year was 2.341 in the liposome-coated group and 0.000 in the standard group. The reported data also shows an average of roughly 9.96 bleeds per year in the liposome-coated group compared with 2.20 in the standard group overall, and that the standard group used a higher average dose of the clotting factor (4,378 units per kg per year) compared with the liposome-coated group (2,524 units per kg per year). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT00882778 · results posted 25 July 2011

  According to the results reported on ClinicalTrials.gov, this trial enrolled 86 participants, all of whom completed the study with no drop-outs. All participants received a treatment called Activated Recombinant Human Factor VII, which is a clotting agent. The trial was measuring whether taking this treatment regularly as a preventive measure (called prophylaxis) changed the number of bleeding episodes participants experienced each month, compared to a period before they started taking it preventively. Results were looked at across different groups: all participants who bled, those who bled frequently, and broken down by age (children under 12, teenagers aged 12–17, and adults aged 18 and over). The reported data shows that, across the overall group of participants who experienced bleeds, there was a reported 45% reduction in the number of bleeds per month when comparing the pre-treatment period to the treatment period. Among those who bled most frequently, the reported reduction was 51%. When broken down by age in this broader bleeding group, children under 12 showed a reported 48% reduction, adults showed a reported 56% reduction, while teenagers showed no reported change (0%). A similar pattern appeared in the frequent-bleeding group, where children showed a reported 56% reduction and adults a reported 49% reduction, but teenagers showed a reported increase of 5%. The reported data also shows differences depending on how often the treatment was given: participants who received doses three times a week showed larger reported reductions (up to 72% in adults) compared to those who received it less than twice a week, where reported changes were much smaller (around 0–6% reduction). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00632814 · results posted 8 February 2011

  According to the results reported on ClinicalTrials.gov, this trial enrolled 32 people in total across three groups, all of whom completed the study. Each group received the same medication — a clotting factor product called rFVIII-FS (Kogenate FS) — but on different schedules: 11 people received one larger dose per week, 13 received two doses per week at different amounts, and 8 received three smaller doses per week. The trial ran for nine months and was primarily measuring how many participants experienced fewer than two joint bleeds (bleeding into a joint) during that time. The reported data shows that, for the main outcome, approximately 72.7% of participants in the once-weekly group, 84.6% in the twice-weekly group, and 75.0% in the three-times-weekly group had fewer than two joint bleeds over the nine months. For the secondary outcomes, the median number of bleeds per person was reported as 3.0, 2.0, and 1.5 for the three groups respectively. The reported data also shows figures for how much medication was used each month on average: approximately 391, 422, and 501 units per kilogram of body weight for the once-, twice-, and three-times-weekly groups respectively. Some additional secondary outcome figures appear in the data but were presented without clear labels distinguishing sub-categories, so those specific breakdowns cannot be described with certainty here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00195442 · results posted 7 February 2011

  According to the results reported on ClinicalTrials.gov, this trial enrolled 288 people who have haemophilia A — a hereditary condition where the blood does not clot properly because of a missing protein called Factor VIII. All participants received a medicine called ReFacto (also known as Moroctocog Alfa), which is a manufactured version of that clotting protein. Of the 288 people who started the trial, 152 completed it and 136 did not finish. The trial tracked how often participants experienced bleeding episodes, how many days they used the medicine, and a number of other measures over time. The reported data shows that, on average, participants experienced approximately 13.4 bleeding episodes per person per year. On the days when bleeding occurred and the medicine was used to treat it, participants used ReFacto on around 20.6 days per year on average. When all treatment days were counted together (including any preventative use), the average came to about 127 days per year per person. The reported average annual amount of the medicine used was approximately 204,500 International Units (IU) — IU is simply the standard way of measuring doses of this type of medicine. The reported data also shows that 668 non-serious unwanted events and 291 serious unwanted events were recorded across all participants during the trial. Additionally, 7 participants were reported to have developed what is called a "de novo inhibitor" — meaning their body appeared to start producing substances that reduced how well the clotting medicine worked. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00289536 · results posted 13 January 2011

  According to the results reported on ClinicalTrials.gov, this trial enrolled 26 participants across three groups who each received a low, medium, or high dose of a Factor VIII product (a protein used in the management of haemophilia). The trial used a crossover design, meaning participants moved through different study periods — in total, across the three periods, between 7 and 10 people were in each dose group at any given time, and nearly all completed every period. The trial was measuring how the body processes Factor VIII at different doses — specifically how quickly levels rise after an infusion, how long the product stays in the body, and how much of it is present over time. The reported data shows that for the primary outcome — how much Factor VIII levels increased per unit of dose given — the figures were 1.7, 1.6, and 1.8 units (IU/dL per IU/kg) for the low, medium, and high dose groups respectively. For the secondary outcomes, the time it took for the amount of Factor VIII in the blood to drop by half (called "half-life") was reported as approximately 11.3, 12.3, and 11.2 hours across the three groups. Other measures tracked how much Factor VIII was present in the blood over the full period after infusion; as expected with higher doses, the total amount recorded over time was larger — rising from around 318 to 616 to 1,116 units across the low, medium, and high dose groups. The reported data shows these were measurements of how the body handled the product at different dose levels, not a comparison against a placebo or another treatment. No information about side effects or safety events was included in the structured results data submitted to ClinicalTrials.gov for this trial. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00759655 · results posted 4 January 2011

  According to the results reported on ClinicalTrials.gov, this trial (NCT00759655) enrolled only one participant in the Xyntha treatment group. Xyntha is a type of clotting factor product used in people with haemophilia A. The trial was set up to measure several things: whether participants developed antibodies (called "inhibitors") that could interfere with the treatment, whether the treatment appeared to have less effect than expected during on-demand use (treating a bleed as it happens) or during preventive (prophylaxis) use, and whether recovery of clotting factor levels in the blood was lower than expected. The reported data shows that the one participant who started the trial did not complete it. For the main question about inhibitor development — where a positive result was defined as a specific antibody level in the blood — the reported figure was 0%, meaning no inhibitor was detected in that participant. However, for all other primary outcome measures (less-than-expected response during on-demand treatment, during preventive treatment, and lower-than-expected recovery), no measurements were reported in the submitted data. Similarly, the secondary outcomes — including the annual rate of bleeds and the number of infusions needed to treat each bleed — also show no reported measurements. Because only one participant was enrolled and that person did not complete the trial, the reported data shows that most of the planned measurements could not be completed or were not submitted to ClinicalTrials.gov. No meaningful conclusions can be drawn from a single, incomplete participant's data, and the absence of numbers for most outcomes means those results were simply not reported. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00866606 · results posted 4 January 2011

  According to the results reported on ClinicalTrials.gov, this trial looked at a clotting factor product called BeneFIX (also referred to as BeneFactor IX) in people with haemophilia B — a condition where the blood lacks enough of a protein called Factor IX (FIX) needed for clotting. Thirty-five participants were enrolled and 31 completed the study. The trial measured how well bleeding episodes responded to BeneFIX infusions, how often a problematic immune response (called an "inhibitor") developed, how many infusions were needed per bleed, and how much of the clotting factor reached the bloodstream after an infusion. The reported data shows that when doctors rated bleeding episode responses on a 4-point scale (where 1 = excellent response and 4 = no response), the average score was 1.70 at 8 hours after an infusion and 1.58 at 24 hours after an infusion. On average, participants required 1.20 infusions to treat each bleeding episode. Regarding the development of inhibitors — where the body's immune system reacts against the treatment — the reported data shows an overall rate of approximately 2.86% across all participants; among those with limited prior treatment history the rate was reported as 4.17%, and among those with more extensive prior treatment history the rate was reported as 0%. No participants were reported as having a "less than expected therapeutic effect," meaning no one was recorded as having no response after two successive infusions within 24 hours for the same bleed. The reported data also shows that the amount of Factor IX reaching the bloodstream after an infusion (called "incremental recovery") was measured at 0.760 IU/dL per IU/kg at the start of the study and 0.727 IU/dL per IU/kg at the six-month mark. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

  View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT00586521 · results posted 19 November 2009

  According to the results reported on ClinicalTrials.gov, this trial involved 20 adults with haemophilia who were given a clotting factor medicine called rFVIII-FS (brand name Kogenate FS). The trial was split into two phases: for the first six months, participants received treatment only when a bleed occurred (called "on-demand" treatment); then, after a short break, they received regular scheduled doses for a further six months to try to prevent bleeds before they happened (called "prophylactic" treatment). The main thing being measured was the number of joint bleeds — bleeds into joints such as knees, ankles, and elbows — in each phase. The reported data shows that during the on-demand phase, the average number of joint bleeds per person was 18.5, while during the prophylactic phase that figure was reported as 1.5. For all bleeds combined (not just joints), the reported averages were 23.7 during on-demand treatment and 1.9 during prophylactic treatment. The trial also measured joint health using a scoring tool called the Gilbert Score, where 0 means normal and higher numbers indicate more joint damage — the reported average score was 25.3 during on-demand treatment and 19.8 during prophylactic treatment. Finally, quality of life was measured using a questionnaire scored from 0 to 100, where higher scores indicate a better outcome; the reported averages were 73.4 during on-demand treatment and 75.9 during prophylactic treatment. No breakdown of these figures by individual participant was reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00243659 · results posted 20 July 2009

  According to the results reported on ClinicalTrials.gov, this trial enrolled 30 people in total — 22 in a "bolus injection" group (where the study treatment was given as a single quick dose) and 8 in a "continuous infusion" group (where it was given as a slow, steady drip). Of those, 20 and 5 participants respectively completed the study. The trial was measuring how well bleeding was controlled during and after surgery in people with haemophilia, using a four-point rating scale: Excellent, Good, Moderate, or None. The reported data shows that, when assessed at the end of surgery, 15 out of 20 bolus injection participants were rated "Excellent" for bleeding control and 5 were rated "Good," with none rated "Moderate" or "None." In the continuous infusion group, 3 out of 5 were rated "Excellent" and 2 were rated "Good," again with none in the lower categories. For the secondary measure — bleeding control assessed at the time of leaving hospital — the reported data shows 18 of the 20 bolus injection participants were rated "Excellent" and 2 were rated "Good." All 5 continuous infusion participants who completed the study were rated "Excellent" at discharge, with none rated lower. It is worth noting that the continuous infusion group was quite small (only 5 completers), so the numbers from that group represent very few individuals. The trial did not report any "Moderate" or "None" ratings in either group at either time point. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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Not medical advice. These are results as reported by the trial sponsor to a public registry, summarised in plain English. They describe what a trial recorded, not whether a treatment is suitable for any person. Verify anything important against the original registry record and discuss what it means for you with your doctor.