What changed for Haemophilia
A plain English summary of what recently moved in the public data Voxsanity tracks for Haemophilia. Updated from government registries. Not medical advice.
How to read these changes: every figure on this page is the difference between two snapshots of what Voxsanity had tracked on each date. Voxsanity only began taking daily snapshots in July 2026 and is still ingesting the public registries, so a rise here is mostly our own coverage expanding rather than new research appearing. Read it as our tracked coverage growing, not as a claim about real-world research changing this fast: it overstates how much actually changed.
Comparing the two most recent snapshots, 27 July 2026 and 28 July 2026.
- Trials recruiting worldwide 90 to 91 (+1)
- Trials on record 865 to 870 (+5)
Newly subsidised on the PBS
No PBS-reviewed medicines are tracked for Haemophilia yet, so new PBS listings cannot be reported for it. This is a gap in what Voxsanity has mapped, not a statement that no medicines are subsidised — check the PBS directly.
Past updates
Tracking began in July 2026. Month on month changes will appear here once a second month of data has been recorded. Until then, the comparison above shows the most recent movement.
Recent trend
Weekly, most recent weeks (fills out to a monthly trend as history grows).
Recent trial results
The 30 most recent of 132 trials tracked for Haemophilia that have reported results to a public registry.
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...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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 ma...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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 treat...
-
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 pa...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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 chan...
-
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 rec...
-
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...
-
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...
-
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 w...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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....
-
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...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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 partic...
-
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 control...
-
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 phas...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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 replaceme...
-
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...
-
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...
-
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...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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. T...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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 occurr...
-
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. Th...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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....
-
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 t...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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 cha...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
-
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...
-
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 show...
-
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 cou...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
See all 132 reported results for Haemophilia
These are the results as reported to the registry, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
What is coming
AI generated pipeline 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
According to NIH funding data, a substantial amount of public research money is currently being directed toward haemophilia, and that investment has been rising year on year. This upward trend is an encouraging signal that the scientific community continues to treat haemophilia as a meaningful research priority. For patients, sustained and growing public funding generally means that researchers ar...
Not medical advice. Voxsanity republishes public government health data in plain English. These figures describe research and registry activity, not the suitability of any treatment for any person. Trial status can change; always verify current status directly with the trial site and confirm Australian availability with your doctor.