Reported trial results for Sickle Cell Disease

133 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

• NCT05714098 · results posted 30 June 2026

  According to the results reported on ClinicalTrials.gov, this trial (NCT05714098) enrolled 30 participants, all of whom completed the study. There was only one group — everyone took part in an exercise program. The trial was designed as a feasibility study, meaning its main goal was not to test whether the exercise program "worked" in a medical sense, but rather to explore whether it was practical to run, whether participants were willing to stick with it, and whether it was acceptable to those taking part. The reported data shows that 25 out of 30 participants completed at least half of the scheduled exercise sessions, which was the main thing the trial set out to measure. On the question of acceptability — that is, whether participants reported enjoying or being comfortable with the program — 29 out of 30 people gave it a score of 8, 9, or 10 out of 10 (where 10 meant "absolute enjoyment"). Regarding adverse events (unexpected health issues that occurred within 48 hours of a session), 3 participants reported at least one moderate or severe adverse event in one reported period, and 0 in another; the data as submitted does not provide further detail to clarify these two figures. The trial also looked at physical measures including walking speed, balance, and aerobic endurance (how far someone could walk in six minutes). The reported data shows that 46% of participants showed a clinically meaningful change in walking speed, 35% in balance, and 57% in how far they could walk in six minutes — though what "clinically meaningful" means in this context was defined by the study team and is not explained further 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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• NCT05407805 · results posted 10 June 2026

  According to the results reported on ClinicalTrials.gov, this trial (NCT05407805) enrolled 98 people with sickle cell disease — 33 in a control group and 65 in a disease-modifying treatment group. The trial was measuring how often participants experienced painful vaso-occlusive crises (VOCs — episodes of severe pain caused by blocked blood flow, a common complication of sickle cell disease). It tracked these events in several ways: through doctor-recorded medical visits, through daily self-reports logged by participants on an electronic app, and through participant-rated scores for pain, tiredness, and ability to do everyday activities. The reported data shows the following numbers. For crises that required a medical visit or contact with a health professional, both groups reported the same annualised rate of approximately 2.02 episodes per year. For self-reported "pain crisis days," the control group reported around 113 days per year and the treatment group around 85 days per year. For self-reported crisis events (counted as separate episodes), the control group reported approximately 16 per year and the treatment group approximately 23 per year. On days when a crisis was occurring, worst pain scores (on a 0–10 scale) were 6.0 for the control group and 6.4 for the treatment group; on non-crisis days, scores were 2.4 and 2.9 respectively. Worst tiredness scores during a crisis were 5.0 (control) and 5.6 (treatment), and on non-crisis days were 3.4 and 3.5. Ability to perform usual physical activities was rated on a 1–4 scale (where higher means more difficulty): during a crisis, scores were 2.3 (control) and 2.4 (treatment); on non-crisis days, scores were 1.3 and 1.4 for both 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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• NCT04426591 · results posted 22 May 2026

  According to the results reported on ClinicalTrials.gov, 22 people took part in this trial, and all 22 completed it. The trial was studying how long transfused red blood cells (RBCs) survive in the body after being given to a participant. To track the cells, researchers used a harmless vitamin-based label called biotin, which acts like a tag so the transfused cells can be identified and counted in blood samples taken over time. A smaller group of 6 participants also took part in an optional add-on activity that looked at whether a special "pathogen-reduced" blood processing technique — designed to reduce the risk of infectious agents in donated blood — affected how long the transfused cells lasted. The reported data shows that, for the main group, the tagged red blood cells were measured at several points after transfusion. At 24 hours, about 97.3% of the transfused cells were still detected compared to the level seen 15 minutes after the transfusion. By around the midpoint of follow-up, that figure had dropped to 80%, and by 12 weeks it had fallen to approximately 21%. The reported data also shows that the "half-life" of the tagged cells — meaning the time it took for roughly half of them to disappear from circulation — was reported as 60 days. A third measurement, called mean potential lifespan (an estimate of how long the cells might have lasted in total), was not reported in the submitted data. For the smaller optional group comparing different types of processed blood, the half-life figures were similar across all three blood types tested: approximately 57.6, 59.2, and 59.3 days respectively, and the estimated time for the cells to fully clear the body was reported as around 115, 104, and 115 days for each 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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• NCT04523376 · results posted 28 April 2026

  According to the results reported on ClinicalTrials.gov, this trial enrolled 8 people in total — 7 with sickle cell disease and 1 with beta thalassemia major. The trial was measuring what happened after participants received a stem cell transplant, specifically looking at whether the transplanted cells successfully "took hold" in the body (called engraftment), whether the body's immune system attacked itself after the transplant (a complication called graft-versus-host disease, or GVHD), and how long it took for white blood cell counts to recover. The reported data shows that in the sickle cell disease group, 2 out of 7 participants experienced graft failure, meaning the transplanted cells did not establish themselves properly. The reported time for white blood cells to begin recovering was 15 days on average for the sickle cell disease group and 13 days for the one person with beta thalassemia major. Regarding GVHD — where the donor immune cells react against the recipient's body — 2 out of 7 participants in the sickle cell disease group developed acute (short-term) GVHD, and 1 developed chronic (longer-lasting) GVHD. No GVHD was reported for the beta thalassemia participant. The reported data also shows that 1 death was recorded in the sickle cell disease group and was reported as being related to the study treatment; no treatment-related deaths were reported in the beta thalassemia group. In terms of participants who completed the study without recorded complications, 5 out of 7 in the sickle cell disease group and the 1 beta thalassemia participant were counted in that 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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• NCT05632354 · results posted 19 March 2026

  According to the results reported on ClinicalTrials.gov, this trial (NCT05632354) tested a medicine called osivelotor at different doses (100 mg, 150 mg, and 200 mg) in people with a blood condition. Participants were split into two broad groups: those who started the medicine straight away, and those who had a delayed start. A total of 47 people began the trial across all five groups, though the data shows that none of the participants were recorded as having completed the study — all 47 were listed as "not completed." The reported data shows several things were measured as primary outcomes. For adverse events (unintended medical occurrences during the trial), the numbers reported were: 14 out of 18 participants in the immediate-start 100 mg group, 13 out of 17 in the immediate-start 150 mg group, 1 out of 1 in the immediate-start 200 mg group, 5 out of 5 in the delayed-start 100 mg group, and 6 out of 6 in the delayed-start 150 mg group experienced a treatment-emergent adverse event. Serious adverse events were reported for 4 participants in the immediate-start 100 mg group, and none in the other groups. The trial also measured changes in hematocrit (the proportion of red blood cells in the blood) and white blood cell counts at 12 and 48 weeks, as well as blood pressure changes. The reported data shows that hematocrit changes at week 12 ranged from −0.3 percentage points (immediate-start 100 mg) to +10.0 percentage points (delayed-start 150 mg), and at week 48 from −9.9 (immediate-start 200 mg) to +5.5 percentage points (delayed-start 150 mg). Blood pressure and white blood cell count changes were also recorded, with figures varying across the dose groups, but no further breakdown or explanatory context 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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• NCT05451940 · results posted 7 January 2026

  According to the results reported on ClinicalTrials.gov, this trial involved 17 participants, all of whom received erythropoietin (a medicine that stimulates the body to produce red blood cells). The trial was measuring whether participants' haemoglobin levels — a measure of red blood cells in the blood — increased after 12 weeks of treatment, and whether the number of blood transfusions they needed changed. One participant did not complete the treatment period, so 16 participants went on to finish the full study including the observation period. The reported data shows that 15 out of 17 participants had a haemoglobin response, meaning their haemoglobin level rose by at least 1.0 g/dL (a unit used to measure haemoglobin concentration) compared to where it started before treatment. For the secondary outcome, the reported data shows an average change of minus 1.1 transfusions per year, meaning that on average participants received approximately one fewer blood transfusion per year during the treatment period compared to the 12 months before the trial began. Several other planned measurements — including heart function tests and exercise capacity — were listed in the trial but no data was reported for those outcomes. 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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• NCT04930445 · results posted 31 December 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04930445) enrolled 265 participants, all of whom were in a single group taking a medicine called Oxbryta (voxelotor). The trial was designed to track changes in several blood measurements over up to 60 months (5 years) after starting Oxbryta. These measurements included haemoglobin (a protein in red blood cells that carries oxygen), reticulocytes (young red blood cells, which can indicate how hard the body is working to make new blood cells), bilirubin (a substance produced when red blood cells break down), and ferritin (a marker of iron stored in the body). Notably, the reported data shows that zero participants were recorded as having completed the study, and all 265 were listed as "not completed." The reported data shows that, compared to each participant's starting level before treatment, haemoglobin levels changed by amounts ranging from approximately −0.2 to +0.7 grams per deciliter across different time points during the study. The percentage of reticulocytes in the blood changed by amounts ranging from about −1.6 to +1.9 percentage points across time points. Bilirubin levels showed small changes, ranging from −0.1 to +0.2 milligrams per deciliter. Ferritin levels — the iron storage marker — showed a wide range of changes across time points, from a decrease of around 248 micrograms per litre to an increase of around 4,513 micrograms per litre at the latest time point. For the MRI-based iron measurement outcome, no data was 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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• NCT04935879 · results posted 2 December 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04935879) enrolled 241 people — 119 in the inclacumab group and 122 in the placebo group. The trial ran for 48 weeks and was looking at vaso-occlusive crises (VOCs) in people with sickle cell disease. VOCs are sudden, severe pain episodes caused by blocked blood flow, serious enough to require a visit to a medical facility or contact with a healthcare provider for strong pain relief. The trial measured how often these crises occurred, how long it took for the first one to happen, and several other related outcomes. The reported data shows that, over the 48-week period, participants in the inclacumab group had an average of 1.49 VOC events, compared with 1.58 events in the placebo group. For how long it took until a first VOC occurred, the reported median time (the midpoint of the group's results, estimated using a standard statistical method) was approximately 28.7 weeks in the inclacumab group and 21.6 weeks in the placebo group. The data for time to a second VOC was not reported for either group. Regarding participants who had no VOCs at all during the 48 weeks, the reported figures were 36.0% in the inclacumab group and 25.7% in the placebo group. For VOCs serious enough to require admission to a healthcare facility with intravenous pain medication, the reported rates were very similar — 0.85 events per 48 weeks for inclacumab and 0.83 for placebo. The reported average number of days spent in hospital due to a VOC was 4.96 days (inclacumab) and 5.37 days (placebo). 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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• NCT03573882 · results posted 18 November 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT03573882) enrolled 179 people with sickle cell disease across three groups. All participants had previously taken part in an earlier trial (NCT03036813), where they had been assigned to either a placebo, a lower dose (900 mg) of voxelotor, or a higher dose (1,500 mg) of voxelotor. In this follow-on study, the trial was primarily measuring how many participants experienced adverse events (unexpected medical occurrences during the study) — both those considered related to sickle cell disease and those that were not. It also tracked how often certain sickle cell-related complications and painful crises occurred over time. The reported data shows that when it came to adverse events linked to sickle cell disease, 47 out of 63 participants from the prior placebo group, 42 out of 58 from the prior 900 mg group, and 42 out of 58 from the prior 1,500 mg group experienced at least one such event. For adverse events not related to sickle cell disease, the reported numbers were 59, 49, and 52 participants respectively. Turning to serious adverse events (those considered more severe or requiring hospitalisation), the reported data shows 39, 33, and 22 participants experienced sickle cell-related serious events across the three groups, while 18, 14, and 15 experienced serious events not related to sickle cell disease. The reported data also shows the rate of all sickle cell-related complications per year was 1.18, 1.08, and 1.05 events per person per year across the three groups, and the rate of painful vaso-occlusive crises (episodes of severe pain caused by blocked blood flow) per year was 1.04, 0.93, and 0.89 respectively. It is worth noting that a relatively small proportion of participants completed the study — 16, 11, and 19 in each group — with the majority not completing it, though the reasons for this are not detailed in the submitted data. No secondary outcome measure data was reported in the structured results available. 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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• NCT04170348 · results posted 24 September 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04170348) enrolled 58 people in total — 27 in a group taking vitamin D3 every day, and 31 in a group taking a larger dose of vitamin D3 once a month. By the end of the study, 22 people in the daily group and 23 in the monthly group had completed it. The trial was primarily measuring how often participants experienced respiratory events (a combined count of chest infections, asthma flare-ups, and a complication called acute chest syndrome) over the course of a year. It also tracked several measures of lung function at the start of the trial and again at 24 months. The reported data shows that for the main outcome — yearly respiratory events — both groups had very similar numbers. The daily vitamin D3 group was reported at 3.3 to 3.4 respiratory events per year, and the monthly group at 3.2 to 3.3 events per year (two sets of figures appear in the data, though the trial registry does not explain the distinction between them). For the lung function measures, the reported data shows results at the start and end of the study. One key lung measure (the total amount of air forcefully breathed out, known as FVC) started at around 86% of the expected normal value in the daily group and 84% in the monthly group, and was reported at 83% and 89% respectively at 24 months. Another measure of how fast air moves out of the lungs (FEV1) started at roughly 85% in both groups, and was reported at 86% and 91% at 24 months. The remaining lung function figures followed a broadly similar pattern across both 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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• NCT05283148 · results posted 24 August 2025

  According to the results reported on ClinicalTrials.gov, this study enrolled 53 participants, all of whom completed the study with no drop-outs. The trial was looking at bone density — essentially how strong and dense the bones are — in people with sickle cell disease (SCD) who experience bone pain. Bone density was measured using a special type of low-dose X-ray scan (called a DXA scan) at three sites: the lower spine (lumbar spine), the hip, and the femoral neck (the upper part of the thigh bone where it meets the hip). Participants were split into two groups, referred to as Group A and Group B. The reported data shows bone density measurements and "Z-scores" for each group. A Z-score is simply a number that shows how a person's bone density compares to what is typical for their age and sex in the general population — a score of zero means average, and a negative score means lower than average. For the lower spine, Group A had a Z-score of −1.35 and Group B had −1.60. For the total hip, Group A's Z-score was −0.75 and Group B's was −0.50. For the femoral neck, Group A recorded −0.95 and Group B recorded −0.60. All Z-scores were in the negative range, meaning both groups measured below the population average, though none reached the threshold of −2.0 or below that is used to flag low bone density. The actual bone density measurements (in grams per square centimetre) were also reported across all three sites for both groups, with Group B generally recording slightly higher values than Group A. 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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• NCT02850406 · results posted 15 August 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT02850406) tested a medicine called voxelotor in children and teenagers with sickle cell disease. It was run in four separate parts, each looking at a different age group or dose level. In total, across all parts, 147 young participants took part — 13 in Part A (ages 6–17, short-term dosing), 40 in Part B (adolescents, up to 28 weeks), 62 in Part C (ages 4–17, up to 52 weeks), and 32 in Part D (ages 6 months to under 4 years, up to 52 weeks). Each part had its own main question it was trying to answer. The reported data shows the following for each part. In **Part A**, the trial measured how much of the medicine got into the blood after a single dose. The peak blood concentration was reported as 24,300 nanograms per millilitre in the 12–17 age group and 47,300 in the 6–11 age group — meaning younger children in this group had roughly twice the peak level in their blood. In **Part B**, the trial measured the change in haemoglobin (a protein in red blood cells that carries oxygen) levels from the start to 24 weeks. The reported average change was +0.7 grams per decilitre in the 900 mg group and +0.2 grams per decilitre in the 1500 mg group. In **Part C**, the trial measured changes in blood flow speed in the brain (measured by an ultrasound technique) from the start to 48 weeks. The reported average change was −0.4 centimetres per second in the 4–11 age group and −26.3 centimetres per second in the 12–17 age group. In **Part D**, which focused on the very youngest children (6 months to under 4 years), the trial recorded how many participants experienced medical events during the study. The reported data shows that all 9 participants in the 6-months-to-under-2 group and all 20 in the 2-to-under-4 group experienced at least one treatment-emergent adverse event (an unwanted medical occurrence that happened or worsened after starting the medicine). Serious adverse events were reported in 6 of 9 and 12 of 20 participants respectively. The data as submitted does not include further breakdown of those events beyond the counts 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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• NCT03938324 · results posted 28 July 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT03938324) enrolled 222 participants in total — 147 in the PiCASO intervention group and 75 in an attention control group (a comparison group that received a different form of contact rather than the main intervention). By the end of the study, 99 participants in the PiCASO group and 53 in the control group had completed the trial. The trial was measuring how people managed their own chronic health conditions over time, looking at things like their knowledge and confidence in managing their health, their readiness to take on more responsibility for their care, their quality of life, and their emotional wellbeing. The reported data shows that scores on all five measures were recorded at multiple points throughout the study for both groups. For the main self-management scale (scored 0–8, where higher is better), the PiCASO group's scores moved from 6.7 to 7.1 across the measurement points, while the control group's scores moved from 6.5 to 6.6. For the patient activation measure (scored 0–100, where higher means more confidence in managing one's own health), the PiCASO group's scores went from 70.0 to 79.8, and the control group's from 68.8 to 76.8. On the transition readiness scale (scored 1–5), the PiCASO group went from 4.1 to 4.4, and the control group from 3.9 to 4.2. For mental health-related quality of life (scored 0–100), the PiCASO group's scores moved from 45.5 to 47.8, while the control group's scores remained at 45.1 across all time points. For emotional distress (scored 0–72, where lower means less distress), the PiCASO group's scores moved from 9.7 down to 8.8, and the control group's from 10.7 down to 10.2. 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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• NCT05445128 · results posted 25 July 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT05445128) was studying a treatment approach involving two investigational drugs — MGTA-145 and plerixafor — given to people with sickle cell disease (SCD). The goal was to mobilise and collect a specific type of stem cell (called CD34+ cells) from the blood, which could potentially be used in future cell or gene therapies. The trial had two planned groups: one receiving the drugs over a single day before a blood collection procedure (called apheresis), and a second group receiving the drugs over two days. According to the results reported on ClinicalTrials.gov, only one participant was enrolled — in the single-day group (Part A) — and no participants were enrolled in the two-day group (Part B). The one participant who started the study also completed it. The reported data shows that for the primary outcomes related to safety monitoring, the one participant in Part A had zero adverse events (unexpected health problems) that led to stopping the study drug, zero serious laboratory test abnormalities (graded as Grade 3 or higher, meaning notably outside normal range), and zero clinically significant changes in vital signs such as blood pressure or heart rate. However, the reported data shows that one participant did have a clinically significant change in at least one blood test or chemistry result from their baseline (starting) measurement. For the primary outcome measuring the actual stem cell collection yield — how many CD34+ cells were collected during apheresis — no numerical result was reported in the submitted data. Similarly, the secondary outcome measuring peak CD34+ cell counts in the blood was also 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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• NCT05561140 · results posted 9 July 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT05561140) enrolled 88 people in total — 45 in the voxelotor group and 43 in the placebo group — all of whom also received standard of care treatment. The trial was looking at leg ulcers in people with sickle cell disease. The main thing it set out to measure was how many participants had their target ulcer(s) fully healed (defined as complete skin regrowth confirmed at two check-ups two weeks apart) by the 12-week mark. All participants later moved into an open-label phase where everyone received voxelotor, though very few completed that later phase. The reported data shows that by week 12, approximately 6.7% of participants in the voxelotor group and 7.0% of participants in the placebo group had their target ulcer(s) fully resolved — meaning very small and similar proportions in each group met that healing milestone. For the secondary outcomes, the reported data shows that the total surface area of target ulcers changed by an average of −4.8 square centimetres in the voxelotor group (a reduction) and +3.4 square centimetres in the placebo group (an increase) by week 12. Regarding new ulcers appearing during the 12 weeks, 24.4% of the voxelotor group and 32.6% of the placebo group were reported to have developed new ulcers. The median time to ulcer resolution was not able to be calculated for either group, as this data was not reported (likely because too few participants reached full resolution). 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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• NCT04218084 · results posted 13 June 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04218084) enrolled 120 children who received voxelotor and 116 who received a placebo (an inactive treatment used for comparison). Participants were children with sickle cell disease whose brain blood-flow speed — measured using a painless ultrasound scan called transcranial Doppler, or TCD — fell in a "conditional" (borderline elevated) range. The trial's main goal was to see whether voxelotor changed that blood-flow speed over 24 weeks. It is worth noting that a relatively large number of participants did not complete the study — 83 in the voxelotor group and 76 in the placebo group. The reported data shows that the primary measurement — brain blood-flow speed — changed from the starting point by an average of −12.06 cm/sec (centimetres per second) in the voxelotor group and −4.29 cm/sec in the placebo group at 24 weeks, meaning both groups showed a reduction, with a larger reduction reported in the voxelotor group. At 48 weeks, the reported figures were −10.33 cm/sec (voxelotor) and −3.86 cm/sec (placebo). For the time it took participants to reach a normal blood-flow reading, the reported data shows a median of 9.3 weeks in the voxelotor group compared with 17.0 weeks in the placebo group. Regarding haemoglobin levels (a measure of red blood cells in the blood), the voxelotor group showed increases of 0.89, 0.79, and 0.08 g/dL at weeks 24, 48, and 96 respectively, while the placebo group showed changes of 0.18, −0.19, and −0.14 g/dL at those same time points. The data for time to conversion to an abnormal blood-flow reading 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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• NCT04028700 · results posted 3 June 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled 8 participants in total — 3 in one group and 5 in the other. It was a crossover study, meaning participants were planned to receive both types of blood transfusion in sequence, with a four-month "washout" period (a break to let the effects of the first transfusion clear) in between. The trial was comparing two types of donated red blood cells: those from donors with a condition called G6PD deficiency (a common inherited difference in red blood cell chemistry) and those from donors without it. The main thing being measured was how well the transfused red blood cells survived in the recipient's body after transfusion. Notably, the data shows that no participants completed the second intervention phase, so only the first round of transfusions appears to have data reported. The reported data shows that, for the primary measure — the percentage of transfused red blood cells still surviving in the body after transfusion — the figure was 82.91% for G6PD deficient red blood cells and 91.03% for non-G6PD deficient red blood cells. For the secondary measure, the trial tracked the mean percentage change in a protein called Haemoglobin A (the normal form of haemoglobin that carries oxygen in the blood). The reported data shows a mean change of 25.87% for the G6PD deficient transfusion group and 21.02% for the non-G6PD deficient transfusion group. Because the second intervention phase shows zero participants, the crossover comparison the trial was designed to make does not appear to have been completed, and full crossover results were 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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• NCT03431285 · results posted 28 May 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled 278 adults who came to an emergency department (ED) with pain. They were split into two groups: 140 people received morphine and 138 received ketamine (a different type of pain-relieving medicine). All participants in both groups completed the study. The trial was measuring pain levels over three hours, how long people stayed in the ED, how much opioid (strong pain medicine) was used, how many people needed to be admitted to hospital, and how many people experienced side effects from the medicines. The reported data shows that pain was rated on a scale of 0 (no pain) to 10 (worst possible pain). Over the three hours, the average pain score reported was 5.6 in the morphine group and 5.7 in the ketamine group. The average time until people were ready to leave the ED was 4.8 hours for the morphine group and 4.7 hours for the ketamine group. When it came to additional opioid medicines used during the ED stay, the morphine group received an average of 0.13 mg per kilogram of body weight, compared to 0.07 mg per kilogram in the ketamine group. The number of people admitted to hospital was 34 in the morphine group and 26 in the ketamine group. The reported data also shows that drug-related side effects — which included things like nausea, vomiting, dizziness, and other reactions — were recorded in 3 participants in the morphine group and 8 participants in the ketamine group. No other outcome figures beyond those listed above were included 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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• NCT03442114 · results posted 25 May 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT03442114) looked at how to support parents of children with sickle cell disease in making decisions about a medication called hydroxyurea. A total of 174 parents took part — 94 were given a tool called the Hydroxyurea Shared Decision Making Toolkit (H-SDM) and 80 received a simpler Clinician Pocket Guide. Of those, 78 and 59 participants respectively completed the study. The trial measured things like how uncertain parents felt about making a treatment decision, how involved they felt in that decision, and how satisfied they were with the process. The reported data shows that on the main measure of "decisional conflict" — a score from 0 (feeling very certain) to 100 (feeling very uncertain) — parents in the H-SDM Toolkit group scored around 19.8 overall, compared to around 22.9 for the Pocket Guide group. Several sub-parts of this scale were also measured, and in each case the H-SDM group's scores were slightly lower (meaning somewhat less uncertainty reported). For how involved parents felt in the decision-making process (scored 0–100), both groups reported similar scores: around 40.3 for the H-SDM group and 39.8 for the Pocket Guide group. On satisfaction with the decision-making process (scored 0–28), both groups also reported similar scores of around 13.2 and 13.5 respectively. The reported data also shows results for a child development screening score and a quality-of-life score related to sickle cell disease, with both groups scoring similarly on each. Regarding whether hydroxyurea was actually offered during the appointment, the data was recorded for participants across three categories — not offered, offered, or previously prescribed — though the numbers varied between groups. These figures were recorded from medical records rather than reported by parents directly. 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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• NCT03806452 · results posted 14 May 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT03806452) looked at a medicine called hydroxycarbamide and compared it to a placebo (a dummy treatment with no active ingredient) in people with kidney disease, specifically measuring changes in protein levels in their urine and kidney function over time. In the first phase of the trial, 46 people received hydroxycarbamide and 40 received the placebo. A smaller group of participants who responded to their initial treatment then continued into a second phase — 18 from the hydroxycarbamide group and 15 from the placebo group. The reported data shows that for the main thing being measured — the number of people whose urine protein level (called ACR, a marker used to assess kidney health) dropped by at least 30% after six months — 18 out of 46 participants in the hydroxycarbamide group and 15 out of 40 in the placebo group reached that threshold. For the secondary measurements, the reported data shows that average kidney function scores (called eGFR, a measure of how well the kidneys filter blood) changed by +3.2 units in the hydroxycarbamide group and −1.0 units in the placebo group. Average ACR values changed by +3.5 units in the hydroxycarbamide group and +1.0 units in the placebo group. Regarding shifts between categories of protein in the urine, 10 hydroxycarbamide participants and 7 placebo participants moved from a mid-level concern (microalbuminuria) to a lower-level concern (normoalbuminuria); 1 hydroxycarbamide and 2 placebo participants moved from a higher-level concern (macroalbuminuria) to the mid-level; and 1 hydroxycarbamide participant versus 0 placebo participants moved from the highest to the lowest category. It is worth noting that a positive change in ACR would typically suggest more protein in the urine rather than less, which may seem at odds with the primary outcome — however, the reported data shows these numbers as submitted to ClinicalTrials.gov, and no further explanation was provided in the structured results to clarify this. 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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• NCT02863068 · results posted 7 May 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled 18 people in total — 10 received a placebo (an inactive treatment) and 8 received a topical sodium nitrite cream applied to their skin ulcers. All 18 participants completed the study. The trial was looking at leg or body ulcers in people with sickle cell disease, and was measuring three main things over 10 weeks: whether methemoglobin levels in the blood changed (methemoglobin is a form of haemoglobin that can rise with certain treatments and is used here as a tolerability marker), whether the total surface area of ulcers changed, and whether participants reported changes in ulcer pain on a 0–10 scale. The reported data shows that methemoglobin levels changed by an average of −0.05% in the placebo group and +0.25% in the sodium nitrite group — both very small shifts from their starting levels. For ulcer surface area, the median change from the start of the study was a reduction of 1.50 cm² in the placebo group and a reduction of 3.26 cm² in the sodium nitrite group. Regarding pain scores, the placebo group reported an average decrease of 0.5 points on the 0–10 scale, while the sodium nitrite group reported an average increase of 0.9 points. The reported data also shows that 5 out of 10 participants in the placebo group and 4 out of 8 in the sodium nitrite group achieved a reduction in ulcer surface area of 25% or more from their starting size. The trial also looked at whether taking a separate medicine called hydroxyurea at the same time made any difference to these results, though the data for those subgroups was not reported in a way that allows straightforward interpretation here. Because this was a small, early-phase study with only 18 participants, 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.

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

• NCT04156906 · results posted 2 May 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled 5 participants, and all 5 completed the study with no one dropping out. The trial was looking at what happens when people who have previously developed an immune response to a blood component called the "D antigen" (known as anti-D) are given red blood cell transfusions that have been carefully matched to their genetic blood type profile. The key question was whether receiving these specially matched blood cells would cause the anti-D immune response to come back, or whether there would be any signs that the transfused red blood cells were being broken down by the body. The reported data shows that out of the 5 participants, 0 showed a reappearance of the anti-D immune response or signs of their body breaking down the transfused red blood cells. In other words, the reported numbers indicate that none of the 5 participants experienced either of those two events that the trial was specifically watching for. No other outcome measures — such as secondary outcomes — appear to have been submitted in the data available on ClinicalTrials.gov, so further results beyond this primary measure were not reported. It is worth keeping in mind that this was a very small study involving only 5 people, which means the reported numbers, while notable, come from a very limited 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.

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

• NCT04906707 · results posted 20 April 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04906707) involved 44 adults in total — 19 in the EaseVRx group and 25 in the Active Control group. The trial looked at a virtual reality (VR) program called EaseVRx for people living with chronic pain. It measured self-reported pain levels at regular intervals over eight weeks, as well as how much participants actually used the VR device (engagement). Most participants completed the trial: 16 out of 19 in the EaseVRx group and 24 out of 25 in the Active Control group. The reported data shows that pain was measured using two different scales. On the daily pain diary scale (scored 0–10, where 0 means no pain and 10 means the worst pain), the EaseVRx group reported average scores of 3.79 in the first four weeks and 2.37 in the second four weeks. The Active Control group reported scores of 3.17 and 2.63 across the same two periods. On a separate monthly questionnaire (scored 0–100), the EaseVRx group's average pain scores went from 66.84 at the start, to 60.28, 56.33, and then 60.00 over the following months. The Active Control group's scores went from 63.07, to 54.06, 48.84, and then 45.96 over the same timepoints. Regarding how much participants used the device, the reported data shows that the Active Control group used the device on more days per week on average than the EaseVRx group across both time periods. Some engagement figures for the Active Control group 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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• NCT03938454 · results posted 10 March 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled 36 adult male participants, all of whom received infusions of a drug called crizanlizumab at a dose of 5 mg/kg. Thirty participants completed the study, while six did not finish. The trial was looking at a condition called priapism — unwanted or painful erections lasting at least 60 minutes — in people with sickle cell disease. The main thing the researchers were measuring was whether the number of these episodes changed over 26 weeks of treatment. The reported data shows that, on average across participants, the number of priapic events fell by 61.3% between the start of the study and the 26-week mark. For the secondary measures, the annualized rate of these episodes (that is, the estimated number of episodes per year, calculated to account for people who left the study early) was reported as 31.1 events per year at baseline and 25.6 events per year during the treatment period. The number of severe, acute episodes — defined as painful erections lasting more than four hours that required an emergency department visit — was reported as 7.0 at baseline, 1.5 at 26 weeks, and 1.0 at 52 weeks. The reported data also shows figures for painful "sickle cell crises" more broadly: uncomplicated pain crises were reported at a rate of 4.1 per year at baseline and 2.5 per year during treatment, while more serious crises were reported at 1.9 per year at baseline and 1.4 per year during treatment. Regarding side effects, the reported data shows that 9 out of 36 participants experienced reactions linked to the infusion process, with smaller numbers of participants reporting individual types of reactions — though the specific breakdown of reaction types was 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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• NCT04419168 · results posted 19 February 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04419168) enrolled 359 adults with sickle cell disease — 181 in a group receiving a computer-based cognitive behavioural therapy program (cCBT) and 178 in a group receiving mobile health education (m-Education). By the end of the study, 125 people in each group had completed it fully. The trial was measuring changes over six months in several areas, including how much pain interfered with daily life, daily pain intensity, mood (depression and anxiety), quality of life, and participants' confidence in managing their condition. The reported data shows that, for the main outcome — a standard pain interference score where higher numbers mean more interference — both groups showed a decrease (meaning less reported interference) over six months. The cCBT group's score dropped by 2.13 points and the m-Education group's dropped by 2.66 points. For daily pain intensity (rated 0–10), the reported data shows a small increase of 0.20 points in the cCBT group and 0.13 points in the m-Education group. On the depression scale (0–27), both groups showed small decreases of 1.33 (cCBT) and 1.12 (m-Education) points. On the anxiety scale (0–21), decreases of 0.85 (cCBT) and 1.49 (m-Education) points were reported. Quality-of-life scores and self-confidence in managing the disease also showed small changes in both groups, with the cCBT group reporting a slightly larger increase in self-confidence (1.45 points) compared to the m-Education group (0.32 points). It is worth noting that these numbers reflect averages across each group, and the data does not indicate what size of change would be considered meaningful in everyday life. Not all participants who started the trial completed it, which is also 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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• NCT04893811 · results posted 16 October 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04893811) looked at a meningococcal B vaccine called Trumenba. It involved two groups: 53 people who received the vaccine as part of this trial (referred to as the B1971060 group), and 51 people whose data came from a previous, similar trial and were used as a historical comparison group (B1971057). Both groups received two doses of the vaccine. The trial measured how the body responded to the vaccine by looking at blood levels of protective antibodies against four strains of the meningococcal B bacteria, and also tracked reactions at the injection site and general body reactions after each dose. The reported data shows that before vaccination, only a small proportion of participants in either group had antibody levels at or above the measurement threshold for each bacterial strain — ranging from roughly 2% to 33% depending on the strain and group. One month after the second dose, these proportions were considerably higher: for the trial group, the reported figures ranged from about 71% to 91% across the four strains, while for the historical comparison group the figures ranged from about 82% to 100%. For injection-site reactions in the seven days after each dose, the most commonly reported reaction was pain, with around 23–27% of the trial group and about 5–12% of the comparison group reporting this after the first dose. General body reactions such as fatigue and headache were also recorded; fatigue was the most commonly reported, with around 55% of the trial group and 51% of the comparison group reporting it after the first dose. 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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• NCT02140554 · results posted 8 October 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT02140554) enrolled 54 participants across three groups — Group A (9 people), Group B (2 people), and Group C (43 people). The trial was measuring the effects of a treatment called lovo-cel, given via an infusion, in people with sickle cell disease. The main focus was on Group C, and the key thing being measured was whether participants experienced a complete stop in painful crisis episodes — known as vaso-occlusive events (VOEs), which are episodes where blood flow is blocked, causing severe pain — during the period between 6 and 18 months after receiving the infusion. The reported data shows that, among the Group C participants who were included in the main analysis, 87.5% had a complete stop in their VOEs during that 6-to-18-month window after the infusion. For a more severe type of these episodes (called severe VOEs), the reported figure was 93.8% achieving complete resolution. The reported data also shows that 86.1% of Group C participants met a measure called "Globin Response" — a combined blood-level target indicating a meaningful change in certain haemoglobin (the protein in red blood cells) levels — and 96.8% of those who had reached that target earlier were still meeting it at the 24-month mark. The average length of time participants maintained that Globin Response was reported as approximately 20.5 months. Finally, the reported data shows that the average number of VOEs per year in Group C went down by 3.50 episodes per year compared to the two years before the trial began. 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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• NCT03593395 · results posted 3 September 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT03593395) enrolled people with sickle cell disease who were moving from childhood to adult healthcare — a process called "transition." A total of 291 participants were enrolled across two groups: 196 people received a structured education-based transition program, and 95 people received that same program plus peer mentoring (support from someone with lived experience of the same condition). By the end of the study, 121 people in the first group and 49 in the second group had completed participation. The reported data shows that the main thing researchers measured was how often participants needed urgent or emergency care, counted as visits per year. The group receiving education only reported an average of 2.46 acute care visits per year, while the group that also had peer mentoring reported an average of 2.96 visits per year. Several other things were also measured. On a quality-of-life scale for young people with sickle cell disease (scored 0–100, where higher is better), the education-only group scored 66.5 at one time point and 68.7 at another, while the peer mentoring group scored 63.4 and 62.4. For perceived social support (also 0–100, higher being better), scores were 76.8 and 75.3 for the education-only group, and 77.8 and 80.6 for the peer mentoring group. On a readiness-for-transition scale (0–88, where a *lower* score means greater readiness), the education-only group scored 10.4 then 9.4, and the peer mentoring group scored 8.5 then 8.0. Participants were also asked how often their healthcare provider explained things in an easy-to-understand way; 48 people in the education-only group and 26 in the peer mentoring group answered "always," while 3 in the education-only group and none in the peer mentoring group answered "usually." Results for some quality-of-care survey categories were not fully broken down in the submitted data beyond participant counts across rating ranges. 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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• NCT01443728 · results posted 9 August 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 62 people in total — 31 in a higher-dose vitamin D3 group (100,000 IU) and 31 in a lower-dose group (12,000 IU). Of those, 28 people in each group completed the study, with 3 in each group not finishing. The trial was measuring whether the dose of vitamin D3 made a difference to the number of breathing-related health events participants experienced, such as respiratory infections, asthma flare-ups, and a condition called acute chest syndrome. It also tracked vitamin D levels in the blood and several measures of lung function over time. The reported data shows that the main outcome — the average number of respiratory events per year — was 3.91 for the higher-dose group and 4.34 for the lower-dose group across one set of measurements, with additional measurement sets reporting 3.34 versus 4.28, and 1.54 versus 1.49 events per year respectively (the trial did not provide labels explaining what each set of measurements corresponds to in the submitted data). For the secondary outcomes, the reported average blood vitamin D level was 36.1 ng/mL in the higher-dose group and 19.1 ng/mL in the lower-dose group. The lung function measurements — which looked at how much air participants could breathe out and how quickly — showed broadly similar figures across both groups at multiple time points, with the reported values sitting in the ranges of roughly 78–86% for one breathing measure (FEV1) and 85–92% for another (FVC), depending on the group and 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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• NCT01891812 · results posted 16 July 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT01891812) enrolled 5 participants who received nitrous oxide (sometimes called "laughing gas") as a form of pain relief. The trial was set up to measure how much pain participants felt right after receiving the nitrous oxide, how long the pain relief lasted before they needed something extra, and whether any participants developed certain side effects — specifically nerve-related symptoms or a type of anaemia (low red blood cell count) linked to low vitamin B12 levels. Notably, the reported data shows that none of the 5 participants completed the trial, meaning all 5 left before it was finished; the reasons for this are not detailed in the reported data. The reported data shows that, on a pain scale of 0 (no pain) to 10 (worst possible pain), participants reported an average score of 5.7 immediately after receiving the nitrous oxide. For the secondary measure, the reported data shows that pain relief lasted an average of 39 minutes before participants needed additional pain medication. For the other pre-specified measures, the reported data shows that 0 out of 5 participants showed signs of nerve-related symptoms at an 8-week follow-up, and 0 participants developed the type of anaemia being monitored. Because no participants completed the trial, these numbers should be interpreted with that important limitation in mind. 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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• NCT04983264 · results posted 10 June 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04983264) tested a study drug called GBT021601 in people with sickle cell disease. The trial was split into three parts: Part A involved a single dose and enrolled 6 participants (all 6 completed); Part B involved multiple doses given over a period of time and enrolled 6 participants (5 completed, 1 did not finish); and Part C was an extended treatment period with 4 participants (3 completed, 1 did not finish). The trial was primarily measuring how the drug affected participants' bodies in terms of health checks — including any unwanted medical events (called adverse events), physical examination findings, blood and urine test results, vital signs such as blood pressure and heart rate, and heart tracing (ECG) results. The reported data shows that across all three parts, unexpected medical events (called treatment-emergent adverse events, or TEAEs) occurred in 1 out of 6 participants in Part A, 3 out of 6 in Part B, and 1 out of 4 in Part C. More serious adverse events (SAEs) were reported for 0, 2, and 0 participants respectively across those three parts. When looking at a broader count of all unwanted events, 5 participants in Part A, 5 in Part B, and 3 in Part C reported at least one such event. No participants in any part had clinically notable findings on physical examination or vital signs checks. One participant in Part A had a clinically notable change in a laboratory test result, and one had a clinically notable ECG finding; none were reported in Parts B or C. The reported data also shows that the drug was measurable in the blood, with peak blood concentration figures of 1.01, 16.2, and 69.2 micrograms per millilitre recorded across the single-dose group in Part A (these figures likely reflect different dose levels, though the data does not label them further). 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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• NCT05506358 · results posted 4 June 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 145 participants across five groups, defined by their haemoglobin type: 33 people with HbSS (a form of sickle cell disease), 45 with HbAS (sickle cell trait), 11 with HbS/β-thalassemia (a related inherited blood condition), 26 with HbA/β-thalassemia, and 30 with HbAA (a comparison group without these conditions). The trial was testing how accurately several low-cost screening tools — including the HemoTypeSC, Sickle SCAN, Gazelle Hb Variant test, a solubility test, and an automated sickling test — could detect sickle haemoglobin and β-thalassemia, compared against a standard laboratory reference method called HPLC. The reported data shows results for three key accuracy measures. The first, **sensitivity** (meaning how often the tests correctly identified people who did have the condition), was reported as: 96.6% for the HbSS group, 100% for the HbAS group, 0% for the HbS/β-thalassemia group, 91.3% for the HbA/β-thalassemia group, and 96.7% for the HbAA group. The second measure, **specificity** (how often the tests correctly identified people who did not have the condition), was reported as: 89.9%, 100%, 99.2%, 99.1%, and 98.1% for those same groups respectively. A third measure — **positive predictive value** (how reliable a positive test result was) — was partially reported, with 71.8% for the HbSS group and 100% for the HbAS group; figures for the remaining groups 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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• NCT04053764 · results posted 4 June 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04053764) enrolled 30 people in the crizanlizumab plus standard care group and 28 people in the standard care only group. The trial was looking at a kidney marker called the albumin-to-creatinine ratio (ACR) — a measure of how much of a protein called albumin leaks into the urine, which doctors use to track kidney health. The main question the trial asked was: after 12 months, how many participants had their ACR level drop by at least 30% from where it started? The reported data shows that at the 12-month mark, 33.3% of participants in the crizanlizumab plus standard care group had at least a 30% reduction in their ACR, compared with 21.4% in the standard care only group. For a secondary measure looking at the same 30% reduction threshold but at 6 months, the reported figures were 30.0% for the crizanlizumab group and 35.7% for the standard care group. The trial also tracked changes in another kidney marker called eGFR (a rough measure of how well the kidneys are filtering), and the reported data shows small percentage decreases in both groups across different time points, with the figures varying between the groups at each check-in. Additionally, a measure of how quickly ACR changed over time (the "slope of decline") was reported as 1.70 mg/g per month for the crizanlizumab group and 4.49 mg/g per month for the standard care 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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• NCT04505969 · results posted 10 May 2024

  According to the results reported on ClinicalTrials.gov, this study involved 192 people across two groups: 108 patients living with sickle cell disease (SCD) — or parents of children with the condition — and 84 healthcare professionals who work in SCD clinics. All 192 participants completed the study, with no dropouts recorded. The study was not testing a medicine or procedure; instead, it was measuring how satisfied patients, parents, and healthcare workers were with the current treatment, care, and clinic services for sickle cell disease. The reported data shows that patients and parents answered questions using a rating scale from 1 to 5, where 1 meant "very dissatisfied" or "very poor" and 5 meant "very satisfied" or "very good." Their average score for satisfaction with current SCD treatment and management was 4.12 out of 5. They rated the overall quality of clinic services at 4.37 out of 5, and their satisfaction with nursing care in the clinic at 4.22 out of 5. For the healthcare professionals, the reported data shows they rated the clinic's facilities for managing SCD patients at 4.12 out of 5, their satisfaction with their own responsibilities in the clinic at 4.22 out of 5, and their overall satisfaction with working with SCD patients at 4.17 out of 5. 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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• NCT04420585 · results posted 7 May 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04420585) enrolled 8 participants in a single treatment group. All participants had both sickle cell disease and bedwetting (nocturnal enuresis). The trial was measuring whether a medication called desmopressin (also known as DDAVP) changed the number of nights on which bedwetting occurred, as well as looking at night-time wakings, daytime tiredness, and day-to-day quality of life. Of the 8 people who started, 5 completed the study and 3 did not. The reported data shows that, on average, the percentage of nights with bedwetting fell by 41 percentage points after about one month on the medication — a negative number here means fewer bedwetting nights were recorded compared to the start of the study. For night-time wakings to use the toilet, the reported data shows an increase of 16.8 percentage points (a positive number indicates more wakings). For daytime tiredness, participants completed a standard questionnaire scored from 10 to 50, and the reported data shows an average change of −6.0 points, where a negative number means a lower tiredness score at one month compared to the start. The quality-of-life outcome (measuring things like activities, feelings, and school performance) was not reported in the submitted data. It is worth noting that only 8 people took part and just 5 finished the study, so the numbers above come from 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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• NCT04579926 · results posted 1 May 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04579926) involved 24 participants — both adolescents with sickle cell disease and their parents — and all 24 completed the study with no dropouts. The trial was testing the "Pinpoint" app, a smartphone application designed for young people living with sickle cell disease, a condition that affects red blood cells and can cause pain and other symptoms. The main thing being measured was how confident the adolescents felt in managing their condition and going about daily life — a concept called "self-efficacy" (essentially, a person's belief in their own ability to cope). A secondary measure looked at how easy and usable participants found the app itself. The reported data shows that adolescent self-efficacy was measured using a 9-question scale with possible scores ranging from 0 to 45, where higher numbers mean greater confidence. Scores were collected at multiple points across the study. According to the results reported on ClinicalTrials.gov, the average scores across those time points were 34.6, 35.8, 37.4, 38.9, and 39.0 — with the full range of individual responses narrowing at later time points (for example, the lowest individual score recorded was 10 at the earliest measurement and 18 at the latest). For the app usability measure, the reported data shows an average score of 72.5 out of 100 on a standard usability scale, where a score above 68 is described as above average. 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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• NCT04587661 · results posted 30 April 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 21 people in total — 11 in a group using an "off-the-shelf" digital Cognitive Behavioural Therapy (CBT) app, and 10 in a group using a version of the app that had been specially adapted. CBT is a type of talking-based mental health support. The trial was measuring how much participants engaged with the app over a four-week period, and also tracked self-reported scores for pain, depressive symptoms, and anxiety before and after the four weeks. All 11 participants in the off-the-shelf group completed the study, while 8 of the 10 in the adapted group completed it. The reported data shows the following engagement figures: participants in the off-the-shelf group opened the app an average of 5.64 times per week, compared to 8.50 times per week in the adapted group. For lessons completed over the four weeks, the off-the-shelf group averaged 1.27 lessons, while the adapted group averaged 7.1 lessons. For text messages sent to a health coach each week, the off-the-shelf group averaged 2.09 messages compared to 6.1 in the adapted group. Regarding the secondary measures, the reported data shows the change in pain score (on a 0–10 scale) from the start to four weeks was 4.2 points for the off-the-shelf group and 3.86 points for the adapted group. The change in depressive symptom score (on a 0–27 scale) was 10.09 points for the off-the-shelf group and 6.57 points for the adapted group. The change in anxiety score (on a 0–21 scale) was 8.91 points for the off-the-shelf group and 3.14 points for the adapted group. These figures represent the difference between each participant's starting score and their score at four weeks, but the data does not report whether scores went up or down from baseline. It is worth noting that this was a very small trial — just 21 people — and no further details about how these numbers should be interpreted were included in the submitted results 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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• NCT03997760 · results posted 23 April 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 19 people in total, split across four groups: 5 received a placebo (an inactive infusion), and the remaining 14 received different doses of an investigational medicine called TAK-755 — either 40, 80, or 160 units per kilogram of body weight (4, 6, and 4 participants respectively). One person in the 40 IU/kg group did not complete the study; everyone else finished. The trial was primarily measuring how many participants experienced medical events (called adverse events) after receiving the infusion, and whether the body's immune system produced antibodies — proteins that can react — against TAK-755. The reported data shows that, for adverse events that started or got worse after the infusion, 2 out of 5 placebo participants, 2 out of 4 in the 40 IU/kg group, 4 out of 6 in the 80 IU/kg group, and 2 out of 4 in the 160 IU/kg group experienced at least one such event. Serious adverse events (those resulting in hospitalisation, being life-threatening, or similarly significant) were reported in 0 placebo participants, 1 participant in the 40 IU/kg group, and 0 in each of the other two TAK-755 groups. Regarding immune reactions, the reported data shows that zero participants in any group developed antibodies against TAK-755, whether pre-existing, newly formed after treatment, or increased after treatment. The reported data also shows results for secondary measures tracking how the drug moved through the body. Peak levels of TAK-755 in the blood appeared to rise with higher doses — for example, peak activity levels were approximately 0.90, 2.01, and 2.95 international units per millilitre for the 40, 80, and 160 IU/kg groups respectively. The time for the drug to reach its peak in the blood was generally under about two hours across all groups. The time for the drug's level to fall by half (called the "half-life") was not able to be calculated for the lowest dose group, but was reported as approximately 55 hours and 42 hours (measured by two different methods) for the 80 IU/kg group, and approximately 65 hours and 47 hours for the 160 IU/kg 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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• NCT03264989 · results posted 23 April 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 57 people with sickle cell disease — 45 received crizanlizumab at a dose of 5.0 mg/kg and 12 received it at a higher dose of 7.5 mg/kg. The trial was primarily measuring how the drug moved through the body over time (known as pharmacokinetics) and how much it blocked a protein called P-selectin (which plays a role in the painful crises associated with sickle cell disease). Secondary measurements looked at the rate of painful vaso-occlusive crises (VOCs) — episodes where blood flow is blocked, causing pain — that led to a healthcare visit or were managed at home. The reported data shows that none of the participants were recorded as having formally "completed" the study, though the reason for this is not explained in the data provided. The reported data shows that, for the 5.0 mg/kg group, the drug reached a peak blood level of 102 μg/mL after the first dose and 123 μg/mL at steady state (when levels in the body have stabilised over time). Pre-dose blood levels — measured just before each injection to check how much drug remained in the body — stayed relatively stable across the dosing period, ranging roughly between about 8.5 and 18 μg/mL. The P-selectin inhibition measurements (a way of tracking how much the drug was blocking that protein) were reported as 33,200 and 66,900 hours × % inhibition after the first and later doses respectively, suggesting the blocking effect was higher after multiple doses, though what this means clinically is not for this summary to interpret. For the secondary outcomes, the reported data shows that in the 5.0 mg/kg group, the annualised rate of VOCs leading to a healthcare visit was 4.00 events per year before treatment and 2.75 during treatment. In the 7.5 mg/kg group, the corresponding figures were 2.00 and 0.97 events per year. For crises managed at home, the reported rate was 0.68 events per participant-year in the 5.0 mg/kg group and 0.71 in the 7.5 mg/kg 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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• NCT03333486 · results posted 2 April 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 31 participants, all of whom received the same treatment combination — a conditioning regimen involving the medicines fludarabine and cyclophosphamide, together with total body irradiation (a type of whole-body radiation) and a stem cell transplant using cells collected from the bloodstream. The trial was measuring how often the disease came back within a year, as well as a number of other outcomes related to the transplant process and participants' health over time. Of the 31 people who started, 29 completed the study and 2 did not. The reported data shows that 14 out of 31 participants had their disease return (relapse) within one year — this was the main thing the trial was set up to measure. For the secondary (additional) measures, the reported data shows that 84% of participants had a successful "engraftment" based on white blood cell counts (meaning the transplanted cells appeared to start producing a type of immune cell called neutrophils), and 77% had successful engraftment based on platelet counts (platelets are the cells that help blood clot). The reported data also shows that roughly 32% of participants (about 1 in 3) experienced a condition called acute graft-versus-host disease, where transplanted cells react against the recipient's body in the short term, and approximately 19% (about 1 in 5) experienced the longer-lasting chronic form of this condition. The reported median overall survival — meaning the point in time by which half of participants were still alive — was 15.7 months, though it is important to note this figure reflects the specific group studied and the timeframe of the 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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• NCT03178643 · results posted 27 March 2024

  According to the results reported on ClinicalTrials.gov, this trial compared three different malaria prevention medicines in a total of 246 participants, split across three groups: 81 people received Proguanil tablets, 83 received a combination called Sulfadoxine/Pyrimethamine-Amodiaquine (SP-AQ), and 82 received a combination called Dihydroartemisinin-Piperaquine (DP). The trial was measuring how often participants developed clinical malaria while taking these medicines, as well as a number of other malaria-related events such as severe illness, hospitalisation, and very low blood counts (severe anaemia). The reported data shows that the main thing being measured — the number of malaria episodes per person per year — differed across the three groups. The Proguanil group recorded 0.04 episodes per person per year, the SP-AQ group recorded 3.05 episodes per person per year, and the DP group recorded 1.36 episodes per person per year. For the secondary measures, the reported data shows that no participants in any of the three groups were recorded as having severe malaria during the study period. Severe anaemia (very low haemoglobin, a measure of red blood cells) was recorded in 8 participants in the Proguanil group, 9 in the SP-AQ group, and 4 in the DP group. Three participants in each group were hospitalised for malaria. Malaria detected under a microscope was recorded in 17, 11, and 19 participants respectively, and suspected but unconfirmed malaria (where treatment was given without a positive test) was recorded in 42, 25, and 40 participants across the three 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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• NCT03634488 · results posted 27 March 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT03634488) looked at whether it was practical and feasible to run a larger study involving children aged 5 to 12 years with sickle cell disease (SCD) — a blood condition — who also had severe malnutrition. The trial took place over 12 weeks and involved three groups: 56 children with SCD who received a ready-to-use therapeutic food (a specially formulated food supplement) combined with a medicine called hydroxyurea; 54 children with SCD who received the food supplement alone; and 22 siblings without SCD who also received the food supplement. Rather than testing whether a treatment cured or prevented anything, the trial was designed to measure practical things like how many eligible children agreed to join, how many stayed until the end, and how consistently participants took the food and medicine as instructed. The reported data shows that out of 111 eligible children with SCD who were approached, 110 agreed to participate, and 1 did not. Of the 22 eligible siblings without SCD, all 22 agreed to join. For the 12-week completion (retention) measure, the reported data shows that only 1 participant in the food-plus-hydroxyurea group did not complete the study, while no participants dropped out in the other two groups. Regarding how well participants stuck to the food supplement, the reported figures show that approximately 99% of the food sachets were returned empty across all three groups — meaning nearly all sachets provided were consumed. No missed clinic visits were recorded for any group. For the hydroxyurea medicine, only about 4.4% of pills were returned unused in the group that received it. The reported data also includes a blood measurement called mean corpuscular volume (MCV) — essentially the average size of red blood cells — which was tracked as an indicator of whether hydroxyurea was being taken as directed. The group receiving food plus hydroxyurea showed an average increase of 5.4 units (fl), while the food-only group showed an average change of −1.0 units. 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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• NCT03716726 · results posted 26 March 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT03716726) looked at a programme called "WE CARE" compared to standard care for families of children with sickle cell disease. A total of 112 people started the trial across both groups — 51 in the WE CARE group and 61 in the standard care group. By the 12-month follow-up point, 31 people in the WE CARE group and 32 in the standard care group had completed the study. The trial was measuring things like visits to the emergency department, whether parents connected with community support services, and how parents were feeling and coping over time. The reported data shows that for emergency department and acute care visits, the WE CARE group recorded 0 visits and the standard care group recorded 1 visit over the study period. For painful episodes related to sickle cell disease that required an emergency or acute care visit, both groups recorded 1 episode each. Regarding prescription coverage for sickle cell medicines, the WE CARE group had prescriptions covering 365 days for both hydroxyurea and penicillin, while the standard care group had coverage of 196 days and 258 days respectively. For the depression questionnaire (a scale of 0–24, where 10 or more suggests major depression), the reported data shows most participants in both groups scored in the lower range, though full individual breakdowns were not clearly separated in the submitted data. For the coping questionnaire, scores across different coping behaviours were reported on a scale of 1–8, with various results across both groups — the data was not reported in a way that allows straightforward plain-language comparison of all individual items. 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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• NCT05315908 · results posted 28 February 2024

  According to the results reported on ClinicalTrials.gov, this trial involved 9,120 people across two groups: 4,489 received an automated phone call and 4,631 received a text message. The trial was looking at whether these two methods of contacting patients could encourage them to get tested for COVID-19, and also tracked whether any tested positive and whether participants received a flu vaccine. It is worth noting that the number of participants recorded as having "completed" the study was very small — only 12 in the automated call group and 25 in the text message group — suggesting the vast majority did not complete the study, though the reported data does not explain why. The reported data shows that 12 people in the automated call group and 25 people in the text message group went on to get a COVID-19 test. Of those who were tested, the reported data shows that zero participants in either group returned a positive COVID-19 result. For the secondary outcomes, 9 people in the automated call group and 21 people in the text message group were reported to have received a flu vaccine. Two other planned measurements — how quickly people came in for testing after being contacted, and how many participants referred household members for testing — were not reported in the submitted results 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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• NCT01850108 · results posted 10 January 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 26 people who received a bone marrow transplant using a "non-myeloablative" (lower-intensity, reduced-chemotherapy) approach from a partially matched family member donor. The trial was designed for people with sickle cell disease or similar blood conditions. Of the 26 who started, 18 completed the study and 8 did not complete it. The trial was measuring a range of outcomes, including deaths related to the transplant procedure itself, complications such as graft-versus-host disease (where donated cells can react against the recipient's body), whether the donated bone marrow successfully took hold, and side effects requiring hospitalisation or other serious medical attention. The reported data shows that zero participants died as a result of the transplant procedure — this was the main (primary) outcome the trial was tracking. For the secondary outcomes, 2 participants were reported as developing acute graft-versus-host disease (graded on a scale from I to IV, where IV is most severe). One participant was reported as having less than 95% of their blood-forming cells coming from the donor at 6 months after transplant, meaning the donated marrow had not fully established itself. Regarding side effects and toxicities (harmful reactions), the reported data shows 1 participant recorded against each of the individual categories measured — including blood-related side effects, non-blood-related side effects, and events serious enough to require hospitalisation — however the data as submitted lists these as separate single-participant counts rather than a combined total, so a definitive overall figure cannot be stated beyond what was 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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• NCT02443545 · results posted 10 January 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 134 participants in total — 89 people who received the study medication, deferiprone, for up to three years, and 45 who received it for up to two years. Of those, 50 and 26 participants respectively completed the study. The trial was measuring changes in iron levels in the liver and heart (using a type of scan called an MRI), as well as a blood marker of iron called serum ferritin, and it was also tracking unwanted events (called adverse events) that occurred during the study. The reported data shows that, looking at all 134 participants together, 104 experienced at least one adverse event of any kind during the study, 35 experienced at least one serious adverse event, and 2 withdrew from the study because of an adverse event. For the secondary measurements, the reported data shows changes in liver iron levels at one, two, and three years of treatment: the average change was −2.64, −3.91, and −6.64 milligrams of iron per gram of liver tissue respectively (negative numbers mean a reduction from the starting level). For the heart MRI measurement, the reported changes were +1.02, +1.00, and +0.98 milliseconds at one, two, and three years. For the blood iron marker (serum ferritin), the reported average changes were −1, −771, and −1,016 micrograms per litre at one, two, and three years respectively. It is worth noting that the trial combined both groups into a single set of numbers for the outcome measures, so the reported data does not separately compare the two groups against each other. 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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• NCT03201874 · results posted 13 December 2023

  According to the results reported on ClinicalTrials.gov, this trial enrolled 111 children and young people with sickle cell disease — 54 in an "Education Control" group and 57 in a "Pain Self-Management Intervention" group. The trial was measuring three main things over about six months: average daily pain levels (rated 0–10), how much pain limited everyday activities (rated 0–100), and how well participants used coping strategies to manage their pain (rated 0–180). Measurements were taken at the start of the trial, after 12 weeks of treatment, and again at 26 weeks (the follow-up point). Most participants completed the trial, with 49 in each group reaching the final follow-up. The reported data shows that at the 26-week follow-up, average daily pain scores were 2.9 out of 10 for the Education Control group and 2.1 out of 10 for the Pain Self-Management group (where a higher number means more pain). For activity limitations, the reported scores at 26 weeks were 12.9 for the Education Control group and 9.0 for the Pain Self-Management group (where a higher number means greater limitations). For coping attempts, scores at 26 weeks were 73.9 for the Education Control group and 85.7 for the Pain Self-Management group (where a higher number means more coping attempts). On the secondary measures, the reported data shows that scores for depressive symptoms and anxiety — measured on a scale where 50 represents the general population average — were broadly similar between the two groups across all time points, generally sitting close to or slightly above 50. Treatment acceptability scores, reported separately for children and parents, were 34.6 and 34.7 respectively out of a possible 45. 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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• NCT04080167 · results posted 22 November 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04080167) looked at a smartphone app called InCharge Health, used by people with Sickle Cell Disease (SCD) and their healthcare providers across seven sites. In total, 382 people started the study across three site groupings — 136 at sites 1 and 2, 99 at sites 3 and 4, and 147 at sites 5, 6, and 7. These included both patients and their healthcare providers. Of those who enrolled, 293 participants were patients and 89 were providers. The trial's main focus was on whether using the app was linked to changes in how consistently patients picked up their prescriptions for a medicine called hydroxyurea, measured over a 24-week period before the app and a 24-week period after. The reported data shows that the primary measure — called "percentage of days covered" (PDC), which tracks how many days out of a given period a patient had their medication available to them — increased by an average of 16.3 percentage points from before the study period to after. The reported data also shows that 240 out of all enrolled patients used the app at least once during the study. For the secondary blood-related measures, the reported numbers show a mean change of 0.70 fL in a red blood cell size measure (MCV), a 1.78 percentage point change in a type of haemoglobin associated with SCD management (fetal haemoglobin), a 0.05 g/dL change in overall haemoglobin concentration, and a −1.48 percentage point change in reticulocyte percentage (an indicator related to red blood cell production). No comparison group data was reported for these measures 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.

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

• NCT04400487 · results posted 21 November 2023

  According to the results reported on ClinicalTrials.gov, this trial enrolled 25 participants who all received a medicine called voxelotor. All 25 completed the initial screening and run-in phases, and 23 of the 25 went on to finish the 24-week treatment period. The trial was measuring physical activity levels in people with sickle cell disease who also had a chronic moderate level of anaemia (low red blood cell count). Physical activity was tracked using a wrist-worn device — similar to a fitness tracker — that recorded movement around the clock. The device's readings were used to calculate overall daily movement, as well as time spent in light and moderate activity. The reported data shows the following changes in physical activity from the start of the trial to later time points. For overall daily movement (measured in "counts per minute," a unit the device uses to record motion), the reported change was −155.6 counts per minute at weeks 10–12, and −79.4 counts per minute at weeks 22–24, meaning the recorded movement figures were lower than at the start of the trial at both time points. For light physical activity, the reported change was +5.8 minutes per day at weeks 10–12 (slightly higher than at the start), and −11.7 minutes per day at weeks 22–24 (lower than at the start). For moderate physical activity, the reported change was −9.0 minutes per day at weeks 10–12, and −5.7 minutes per day at weeks 22–24, both lower than the starting figures. No comparison group (such as a placebo group) was included in this trial, so these figures reflect changes within the one group of participants 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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• NCT04474314 · results posted 18 October 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT04474314) looked at a medicine called IMR-687 in people with sickle cell disease — a condition where abnormal red blood cells can block blood vessels, causing painful episodes called vaso-occlusive crises (VOCs). A total of 115 people took part: 49 received a higher dose of IMR-687, 34 received a lower dose, and 32 received a placebo (a dummy treatment with no active ingredient). The trial measured two main things: how often these painful crises occurred, and how many participants experienced unwanted health events (called adverse events). The reported data shows that the yearly rate of painful crises — calculated by working out roughly how many episodes each person had per year while on the treatment — was 2.32 for the higher-dose group, 1.20 for the lower-dose group, and 2.48 for the placebo group. Regarding unwanted health events, the reported data shows that 40 out of 49 people in the higher-dose group, 29 out of 34 in the lower-dose group, and 28 out of 32 in the placebo group experienced at least one adverse event. More serious adverse events were reported in 17 people in the higher-dose group, 12 in the lower-dose group, and 12 in the placebo group. It is also worth noting that the vast majority of participants did not complete the study — only 2, 6, and 8 people finished in each group respectively — though the reasons for this were not detailed 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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• NCT02757885 · results posted 12 October 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT02757885) enrolled 10 people who received a bone marrow transplant as a treatment for sickle cell disease. The trial was measuring how well the donated bone marrow "took hold" in recipients' bodies, whether sickle cell disease came back, and whether participants survived. Of the 10 who started, 6 completed the study and 4 did not complete it (the reason for non-completion was not reported in the data provided). The reported data shows that, out of the 10 participants, 5 achieved what the trial called "event-free survival" — meaning they were alive with the donor's bone marrow working steadily and no new signs of sickle cell disease returning. One participant experienced what is called a "primary graft rejection," meaning the donor cells were not detected in the blood by day 42 after the transplant. No participants experienced a "late graft rejection" (where donor cells initially appeared but then disappeared after day 42). One participant had the sickle cell disease return. For overall survival — simply being alive, whether or not sickle cell disease had returned — the reported data shows 6 out of 10 participants were alive at the end of the study. Additionally, 5 participants showed successful engraftment of white blood cells (neutrophils) and platelets, meaning the donated bone marrow appeared to be producing these blood cells. 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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• NCT03733249 · results posted 26 September 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03733249) enrolled 184 participants in total. The study looked at two treatments used together — rivogenlecleucel (also called BPX-501, a type of modified immune cell therapy given after a stem cell transplant) and rimiducid (a drug that can be used to manage serious side effects of the immune cell therapy). Of the 184 people who started, 171 received rivogenlecleucel, 142 were included in the main analysis group (called the "Intent-to-Treat" population), and 16 received at least one dose of rimiducid. The trial measured how many participants survived overall, how many remained free of their original disease, and how many avoided a relapse (return of illness) — all tracked at one and two years after treatment. The reported data shows the following numbers from the main analysis group, which was split into two subgroups: those who had a transplant because of a cancer-related ("malignant") condition, and those who had one for a non-cancer ("non-malignant") condition. For overall survival, the reported data shows that in the malignant group, 51 participants were counted at the one-year point and 50 at the two-year point, while in the non-malignant group those figures were 69 and 63 respectively. For disease-free survival (staying free of the original non-cancer condition), the non-malignant group had 69 participants counted at one year and 63 at two years. For relapse-free survival in the malignant group (not experiencing a return of cancer), the reported data shows 50 participants at one year and 48 at two years. Additional smaller numbers reported alongside these figures appear to relate to specific events or subsets within each timepoint, though the data as submitted does not provide enough labelling detail to describe each of those figures precisely. 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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• NCT02090881 · results posted 14 August 2023

  According to the results reported on ClinicalTrials.gov, this trial involved 25 children aged 2 to 15 years who had sickle cell disease (SCD). All 25 children who started the study completed it. The trial was measuring blood flow speed in a major brain artery — the middle cerebral artery — using two different ultrasound machines, to see whether both machines produced the same readings. This type of measurement is used as part of a screening programme to assess stroke risk in children with sickle cell disease. The reported data shows that the key measurement used was called the "Time Averaged Mean of the Maximum" (TAMM) velocity — simply put, this is an average of the peak blood flow speed recorded in the artery, measured in centimetres per second. According to the results reported on ClinicalTrials.gov, both ultrasound machines — a Philips IU-22 and a Zonare Z-One — recorded an average TAMM value of 120 cm/sec in the middle cerebral artery for this group of children. The trial also compared how each machine categorised stroke risk, though the detailed breakdown of those categorisations was not reported in the structured results data 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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• NCT03285178 · results posted 21 July 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03285178) tested a medicine called olinciguat (also known as IW-1701) at four different daily doses — 2 mg, 4 mg, 6 mg, and 18 mg — compared against a placebo (a dummy pill with no active ingredient). A total of 88 people entered an initial run-in phase where everyone received a placebo, and 70 of those went on to the main double-blind treatment phase, where neither participants nor researchers knew who was receiving which treatment. The main things the trial set out to measure were treatment-emergent adverse events — that is, any unwanted medical occurrences that happened after participants started taking the study drug. The reported data shows that across all groups in the double-blind phase, the number of participants who experienced at least one such event varied by group. In the two placebo groups, 6 out of 10 and 6 out of 9 participants reported at least one adverse event. In the olinciguat groups, the numbers were 5 out of 8 (2 mg), 5 out of 8 (4 mg), 8 out of 11 (6 mg), and 21 out of 24 (18 mg). The total number of individual adverse event occurrences recorded was 26 and 29 in the two placebo groups, and 36, 30, 39, and 101 in the four olinciguat dose groups respectively. The reported data also shows that the number of participants whose adverse events were considered possibly related to the study drug was 3 and 0 in the placebo groups, and 2, 0, 4, and 7 across the four olinciguat dose groups. The reported data also shows that when adverse events were grouped by how serious they were, the most severe events (graded "moderate" or above) occurred in 0 placebo-1 participants, 4 placebo-2 participants, 0 in the 2 mg group, 2 in the 4 mg group, 2 in the 6 mg group, and 6 in the 18 mg group. No data appears to have been reported for deaths or life-threatening events specifically broken out in this dataset. 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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• NCT02633397 · results posted 19 July 2023

  According to the results reported on ClinicalTrials.gov, this trial looked at a medicine called riociguat in people with sickle cell disease — a condition that affects red blood cells. A total of 130 people took part: 66 received riociguat and 64 received a placebo (a dummy treatment with no active ingredient). The trial's main focus was on measuring how many participants experienced a serious adverse event (a significant medical problem) during the study period. Secondary measurements included painful sickle cell crises, general adverse events, pain intensity scores, and how far participants could walk in six minutes. The reported data shows that, for the main outcome, 15 out of 66 people in the riociguat group and 20 out of 64 people in the placebo group experienced at least one serious adverse event. For the secondary outcomes, 11 people in the riociguat group and 14 in the placebo group had a serious adverse event specifically related to a sickle cell painful crisis. When looking at all adverse events (not just serious ones), 53 people in the riociguat group and 45 in the placebo group reported at least one. Sickle cell complications during the 12-week study period were recorded in 23 people taking riociguat and 21 taking placebo. Pain scores (on a scale of 0–10, where 0 means no pain) averaged 3.18 for the riociguat group and 3.32 for the placebo group at 12 weeks. The six-minute walk distance averaged 391.97 metres for the riociguat group and 431.49 metres for the placebo 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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• NCT03933397 · results posted 3 July 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03933397) enrolled 328 people in total — 162 in a group receiving a pain medication plan tailored to the individual patient (called the "Patient-Specific Protocol") and 166 in a group receiving a plan based on the patient's body weight (the "Weight-based Protocol"). The trial was looking at pain management during emergency department visits, most likely for a condition causing severe pain episodes. Of those who started, 50 people in the first group and 46 in the second group completed the trial, with the majority not completing it — the reasons for this were not detailed in the reported data. The reported data shows that the main thing being measured was the change in a person's pain score during their emergency department visit, rated on a scale of 0 (no pain) to 100 (worst pain imaginable). In the Patient-Specific Protocol group, pain scores dropped on average by 27.0 points, while in the Weight-based Protocol group they dropped by 27.6 points — a very similar result between the two groups. For secondary measurements, the reported data shows the average time spent in the emergency department was 3.4 hours for the Patient-Specific group and 4.2 hours for the Weight-based group. The average time from being seen to receiving the last medication dose was 2.5 hours and 2.6 hours respectively. Within 7 days after the visit, 11 hospitalisations were recorded in the Patient-Specific group and 14 in the Weight-based group. The number of participants who experienced side effects was reported across several categories, with figures ranging from 4 to 36 participants depending on the type of side effect — however, the specific names of each side effect category were not included in the data provided, so a detailed breakdown cannot be given 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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• NCT01389024 · results posted 28 December 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT01389024) enrolled 12 participants in total, split evenly into two groups — 6 people received a medicine called hydroxyurea, and 6 received a placebo (a dummy treatment with no active ingredient). The trial was measuring whether participants experienced certain brain-related complications, specifically unusual blood flow in the brain (detected by a type of ultrasound scan), silent brain injury, or stroke. Of the 12 who started, 10 completed the study — one person in each group did not finish. The reported data shows that, when looking at the primary outcome — brain-related complications — 1 out of 6 participants in the hydroxyurea group experienced one of these events, compared with 4 out of 6 participants in the placebo group. These are simply the numbers as recorded; the trial does not draw broader conclusions from them on its own. The reported data also shows results for two secondary outcomes related to MRI scans (brain imaging procedures, some of which required sedation to keep participants still). Out of all MRI scans performed across the study, 3 resulted in serious adverse events (meaning unexpected medical events considered significant enough to be formally recorded). The same 3 serious adverse events were also linked specifically to MRI scans that required sedation. It is worth noting that the trial was very small, and the data was not reported separately by treatment group for these MRI-related outcomes. 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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• NCT01877837 · results posted 7 November 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT01877837) enrolled 30 people with sickle cell anaemia. Of those, 25 completed the study and 5 did not finish. The trial was looking at two main things after a stem cell transplant: how many participants experienced "graft failure" (meaning the transplanted cells did not take hold in the body as intended) within two years, and how many participants were still alive at the end of the two-year follow-up period. The reported data shows that 3 out of 30 participants experienced graft failure. For overall survival — meaning the number of participants who were alive at the end of the study period — the reported figure was 23 out of 30 participants. No other outcome figures were included in the submitted results 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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• NCT02372877 · results posted 29 July 2022

  According to the results reported on ClinicalTrials.gov, this trial enrolled 83 people with sickle cell disease (SCD) who underwent a red blood cell exchange or depletion/exchange procedure using a medical device called the Amicus system. Of those 83 participants, 59 completed the study and 24 did not complete it. The trial was primarily measuring how closely the procedure could hit a pre-set target for replacing a patient's sickle red blood cells with donor red blood cells — essentially, how accurate the machine was at reaching the intended goal. The reported data shows that the main measurement — a ratio comparing the actual result of the cell exchange to the intended target — came out at 0.978. A ratio of 1.0 would mean a perfect match to the target, and the trial had pre-defined an acceptable range of 0.75 to 1.25, so the reported figure fell within that range. For a secondary measurement looking at a similar accuracy check on the patient's blood thickness (called haematocrit) at the end of the procedure, the reported ratio was 1.19, which also fell within that pre-defined range. The reported data also shows that after the procedure, patients had on average about 27% fewer white blood cells and about 49% fewer platelets (the tiny cells that help blood clot) compared to before. Regarding serious problems linked to the device itself, the reported number was zero device-related serious adverse events recorded during or shortly after the 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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• NCT02536170 · results posted 6 June 2022

  According to the results reported on ClinicalTrials.gov, this trial enrolled 108 people in total — 36 in each of three groups. All 108 participants completed the study, with no dropouts recorded. The trial was looking at whether L-Arginine (an amino acid supplement), given either as a standard dose or as a higher "loading" starting dose followed by a regular dose, made any difference compared to a placebo (a dummy treatment) in people experiencing a sickle cell pain crisis — a painful episode caused by blocked blood flow. The main thing being measured was the total amount of opioid (strong pain relief) medication each group needed during their time in the emergency department and hospital. The reported data shows that participants in the standard L-Arginine group received an average of 1.77 mg per kilogram of body weight in opioid medication, the loading dose group received 1.95 mg/kg, and the placebo group received 2.36 mg/kg. For hospital stay length, the reported data shows the standard L-Arginine group spent an average of 71.3 hours in hospital, the loading dose group 76.9 hours, and the placebo group 84.9 hours. The time until participants felt well enough to switch from injected to oral (tablet) pain relief was reported as 57.3 hours for the standard L-Arginine group, 55.1 hours for the loading dose group, and 69.3 hours for the placebo group. For the time spent in the emergency department, the figures reported were 6.29 hours, 5.67 hours, and 4.08 hours respectively. Pain scores (rated 0–10, where 10 is the worst pain imaginable) at the start of the study were similar across all three groups (around 8.86–8.89), and at the end were also similar (4.5, 4.75, and 4.56). The data for one secondary measure — time to pain crisis resolution specifically within the emergency department — was 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.

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

• NCT01590628 · results posted 27 April 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT01590628) looked at a cord blood transplant product called NiCord, which is a specially processed form of donated umbilical cord blood used in bone marrow transplantation. A total of 16 people took part — 13 received NiCord together with a second, unprocessed cord blood unit, and 3 received NiCord on its own. The trial was primarily measuring two things: any immediate reactions during or shortly after the infusion, and whether the transplanted cells successfully "engrafted" (meaning the new blood cells began growing) within 42 days. The reported data shows that, for immediate infusion reactions within 24 hours, zero participants in either group experienced a reaction classified as acute toxicity at the first measurement point. At a second measurement point, 3 participants in the NiCord plus unprocessed cord blood group had a recorded event, while zero were recorded in the NiCord-alone group. For engraftment — defined as the body producing enough new white blood cells to reach a certain level for three days in a row — all 13 participants in the combined group and all 3 in the NiCord-alone group met this measure. On the secondary outcomes, the reported data shows that transplant-related deaths were recorded as 0% in both groups. Event-free survival at 100 days (meaning alive with the transplant still working) was reported as 92.3% in the combined group and 66.6% in the NiCord-alone group. Overall survival at 180 days was reported as 92.3% for the combined group and 100% for the NiCord-alone group. It is worth noting that this was a very small trial with only 16 participants across both groups, and the numbers 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.

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

• NCT04156399 · results posted 12 April 2022

  According to the results reported on ClinicalTrials.gov, this was a small pilot trial (NCT04156399) that enrolled 6 adults with sickle cell disease (SCD) — a blood condition that can cause ongoing pain. All participants were in a single group that received acupuncture for chronic pain. The trial was not designed to test whether acupuncture reduced pain; instead, it was designed to find out two things: whether it was practical to run this kind of study with SCD patients (called "feasibility"), and whether participants found the study process acceptable. The reported data shows that all 6 eligible participants agreed to join and started the study, which the researchers described as 100% recruitment of eligible patients. Of the 6 who started, 2 fully completed all 10 acupuncture sessions. Three others completed 8 or 9 sessions before the study was halted due to COVID-19, and the researchers noted these participants had not missed any scheduled sessions and had completed all required questionnaires before and after — giving a reported retention figure of 83%. The sixth participant was unable to finish due to hospitalisation. For acceptability, participants completed a 10-question survey scored out of 20 (where higher scores reflect greater satisfaction with the study and acupuncture experience). The reported average score was 16.4 out of 20. The researchers had set a benchmark of 80% of participants scoring at least 80% of possible points to consider acceptability high, though the data as reported does not break down individual scores further. It is worth noting this was a very small pilot study — only 6 people — and it was cut short by the COVID-19 pandemic, which the researchers acknowledged affected completion. 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 ↗

• NCT03825341 · results posted 18 March 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT03825341) was set up to compare a liquid form of a medicine called hydroxyurea with the standard capsule form in infants aged 9 months to under 2 years. The trial had two groups: one receiving the liquid hydroxyurea and one receiving the capsule. The study was measuring how the body absorbed and processed the liquid formulation — for example, how quickly the medicine reached its highest level in the blood, how long it stayed in the body, and how fast the body cleared it. The reported data shows that only one participant was enrolled in total — one person in the capsule group — and that person did not complete the study. No participants were enrolled in the liquid hydroxyurea group at all. Because of this, the reported data shows no results for any of the primary outcome measures. All the measurements that were planned — including peak blood levels, absorption over time, and clearance rates — were listed as not having any data reported. In short, according to the results reported on ClinicalTrials.gov, the trial did not gather enough participants to produce any outcome results, and all primary outcome figures are absent from 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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• NCT01044901 · results posted 24 September 2021

  According to the results reported on ClinicalTrials.gov, this trial enrolled 51 people in total — 38 people living with Sickle Cell Disease (SCD) and 13 healthy volunteers. All 51 participants completed the study with no drop-outs. The trial was an observational study, not a treatment trial. It used MRI (magnetic resonance imaging) scans to measure the size and function of the heart's chambers in people with SCD, with the healthy volunteers providing a comparison ("normal range") group. The goal was to better understand how SCD affects the heart and lungs, particularly in relation to raised blood pressure in the lungs and the heart's ability to relax and fill properly. The reported data shows a series of heart measurements taken from the MRI scans. The left ventricle (the heart's main pumping chamber) had an average end-of-fill volume of 124.0 mL/cm² in SCD participants, compared with 78.7 mL/cm² in healthy volunteers. The left ventricle's end-of-pump volume was reported as 47 mL/cm² in the SCD group versus 31 mL/cm² in healthy volunteers. The left ventricle's muscle mass index was 77.2 g/cm² in the SCD group and 51.6 g/cm² in healthy volunteers. For the right ventricle (the chamber that pumps blood to the lungs), the end-of-fill volume was 126.4 mL/cm² in SCD participants versus 83.0 mL/cm² in healthy volunteers, and the end-of-pump volume was 56.3 mL/cm² versus 37.8 mL/cm². Finally, the size of the left atrium (the chamber that receives blood returning from the lungs) was reported as 64.8 mL/cm² in the SCD group, compared with 41.1 mL/cm² in healthy volunteers. The reported data shows that across every heart measurement taken, the figures for people with SCD were numerically higher than those recorded for healthy volunteers. What those differences mean clinically is a matter for medical interpretation, and no further detail — such as the range of individual results or measures of statistical certainty — was included in the data 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.

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

• NCT01054768 · results posted 3 August 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT01054768) involved 37 people in total — 17 in the group receiving a combination of Alpha-lipoic Acid and Acetyl-L-carnitine (two dietary supplements), and 20 in the placebo group (a dummy treatment). The trial was measuring C-Reactive Protein (CRP), a substance found in the blood that tends to rise when there is inflammation in the body. Not everyone who started the trial finished it: 9 out of 17 people completed the supplement group, and 17 out of 20 completed the placebo group. The reported data shows that, at the end of the study, the average CRP level in the supplement group was 11.4 mg/L (milligrams per litre of blood), while the average CRP level in the placebo group was 6.8 mg/L. These are the figures as submitted by the study sponsor. It is worth noting that no additional outcome measures — such as whether the difference between these two numbers was considered statistically meaningful (that is, unlikely to be due to chance) — were reported in the structured data on ClinicalTrials.gov. The reported data also does not include information about side effects or other health outcomes, so no conclusions about broader impacts on participants can be drawn from what was submitted. The relatively high number of people who did not complete the supplement group (8 out of 17) is noted in the data, but no explanation for this was provided 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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• NCT03694548 · results posted 16 July 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03694548) was set up in two parts to explore whether a yoga programme was a practical option for teenagers with sickle cell disease (SCD) who experience ongoing pain. Part A involved a survey: 18 teenage patients and 17 of their parents were approached to complete questions about their attitudes and experience with yoga. Part B was a structured yoga programme made up of 8 in-person sessions, which was open to teenagers who had completed the survey. The trial was not primarily testing whether yoga reduced pain — it was measuring whether enough people would agree to take part and follow through, which are important steps before running a larger study. The reported data shows that of the 18 teenagers who started the Part A survey, 15 completed it (3 did not finish). Of those, 12 agreed to go on and join the yoga programme in Part B. However, only 1 of the 12 people who started the yoga programme completed it — 11 did not finish. Because almost no one completed the yoga programme, the reported numbers for several key measures in Part B all came back as zero: no participants attended at least 6 of the 8 yoga sessions, no participants completed all the study assessments before and after the programme, and no participants submitted the required number of pain diary entries. The reported data also shows that zero participants had an emergency department visit or hospital stay for pain within 24 hours of a yoga session. The number of adolescents approached who consented to the Part A survey was not reported in the data submitted to ClinicalTrials.gov. The reported data shows that while a reasonable number of teenagers agreed to take part in the survey stage, very few were able to complete the yoga programme itself, meaning most of the targets the study set out to measure were not met. The reasons for people not finishing the programme were not detailed in the data 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.

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

• NCT03020615 · results posted 25 June 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03020615) enrolled a total of 58 people. Most participants were randomly assigned to one of two groups — 26 to a "Stable Dosing" group and 25 to an "Intensive Dosing" group — while 7 others took part in a separate, non-randomised group. The trial was measuring things like how many people could be enrolled and kept on their medication, and whether a specific type of haemoglobin (a protein in red blood cells) called fetal haemoglobin could be measured at the start and end of the study. This kind of trial is focused on testing whether the study itself can be run successfully, rather than testing a treatment outcome directly. The reported data shows that, of the 51 people who were randomly assigned to the two dosing groups, 41 took their ongoing medication at least 80% of the time — meaning they had access to their medicine for at least 80% of the days they were supposed to. The reported data also shows that fetal haemoglobin levels were successfully collected at both the beginning and end of the study for 19 people in the Stable Dosing group and 23 in the Intensive Dosing group. Regarding hospital stays, 2 participants in each of the two dosing groups were reported to have had a hospitalisation during the study. The trial also tracked reasons why some people declined to take part — 33 people gave the most common reason, with smaller numbers giving other reasons, though the specific reasons behind each count were not detailed 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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• NCT01048905 · results posted 10 June 2021

  According to the results reported on ClinicalTrials.gov, this trial enrolled 13 participants, all of whom received L-glutamine (an amino acid supplement). Twelve participants completed the study, while one did not finish. The trial was measuring several things after 8 weeks of treatment, with the main focus being a specific ratio in red blood cells — the glutamine-to-glutamate ratio — which researchers used as a marker of a type of cell stress called oxidative stress. A number of secondary measurements were also taken, including levels of glutamine in the blood, heart function using an ultrasound test, how far participants could walk in six minutes, and standard blood tests checking liver and kidney function. The reported data shows that the main measurement — the red blood cell glutamine-to-glutamate ratio — was reported as 2.40 at 8 weeks. For the secondary measurements, the blood glutamine level was reported as 685.8 µmol/L (micromoles per litre, a standard unit for measuring substances in blood). A heart ultrasound measurement called tricuspid regurgitant jet velocity (which reflects blood flow through one of the heart's valves) was reported as 2.8 metres per second. The reported data shows that participants walked an average of 366 metres in the six-minute walk test. Liver function readings were reported as 38 and 63 U/L (units per litre) for two different liver enzymes, and kidney function readings were reported as 0.9 and 17 mg/dL (milligrams per decilitre) for creatinine and blood urea nitrogen respectively. It is worth noting that this trial had no comparison group, so these figures represent results from the treated group only, with no separate untreated group 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.

  View reported results on ClinicalTrials.gov ↗

• NCT03615924 · results posted 9 April 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03615924) enrolled 193 participants in total — 101 received ticagrelor (at one of three dose levels: 15, 30, or 45 mg twice daily) and 92 received a placebo (an inactive dummy treatment). The trial was measuring events related to sickle cell disease, specifically "vaso-occlusive crises" (VOCs) — episodes where blood flow is blocked, causing severe pain or a serious lung complication called acute chest syndrome. It is worth noting that the data shows zero participants were recorded as having formally "completed" the study, and all participants were listed under "not completed," though no further explanation for this is provided in the submitted data. The reported data shows that across the whole treatment period, participants in the ticagrelor group had a total of 249 VOC events (the primary thing being measured), compared with 202 VOC events in the placebo group. For the secondary measurements: painful crisis episodes totalled 248 in the ticagrelor group versus 209 in the placebo group; acute chest syndrome events were 6 in each group. The total number of days spent in painful crisis was 1,476 in the ticagrelor group and 1,441 in the placebo group. VOC events that required a hospital or emergency department visit numbered 87 in the ticagrelor group compared with 51 in the placebo group, and the total number of days spent in hospital for VOC events was 526 in the ticagrelor group versus 256 in the placebo group. These are raw totals across all participants in each group; no adjusted or per-person figures were included 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.

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

• NCT03541980 · results posted 2 April 2021

  According to the results reported on ClinicalTrials.gov, this trial enrolled 71 people in total — 35 in the intervention group and 36 in the placebo group. All 71 participants completed the study, with no drop-outs recorded. The trial was measuring how much opioid pain medication people needed after an initial assessment, as well as their pain levels, whether they needed to be admitted to hospital, any unwanted side effects, and how satisfied they were with their pain management. The reported data shows that both groups received the same average amount of opioid medication — 0.2 mg per kilogram of body weight. Pain scores, measured on a scale of 1 to 10 where higher numbers mean more pain, were reported as 5.5 for the intervention group and 5.2 for the placebo group. The reported data shows that 25 participants in the intervention group and 21 in the placebo group went on to be admitted to hospital for further pain management given through a drip or injection. When it came to unwanted side effects from the medications, zero participants in either group were recorded as experiencing any. Satisfaction with pain management was reported as 84% in the intervention group and 85% in the placebo 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.

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

• NCT02851615 · results posted 17 March 2021

  According to the results reported on ClinicalTrials.gov, this trial involved 58 young people with a chronic health condition — 27 in a group called "SCThrive" and 31 in an "Attention Control" group (a comparison group that received a different type of support). The trial was measuring things like how ready and confident participants felt about managing their own health, their self-management skills, and their knowledge about their condition. These were measured at the start of the trial and again after six weeks. The reported data shows that for the main outcome — a score measuring health-related confidence and readiness for self-management (rated on a scale of 0 to 100, where higher means more active involvement in one's own health) — the SCThrive group started with an average score of 68.48 and finished at 76.57, while the Attention Control group started at 69.13 and finished at 68.82. For a secondary measure looking at practical self-management skills (scored 1 to 5, where higher is better), the SCThrive group went from 3.46 to 3.68, while the Attention Control group went from 3.54 to 3.53. A third measure, which used an interview to assess knowledge and skills across areas like medications and nutrition (scored out of 6), was only reported for the SCThrive group — their average score went from 1.19 at the start to 1.15 at six weeks. No data for the Attention Control group on this measure was 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.

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

• NCT03387033 · results posted 1 March 2021

  According to the results reported on ClinicalTrials.gov, this trial involved 20 participants who all completed the study — none dropped out. The trial was looking at whether a virtual reality (VR) session was feasible to use, primarily by asking participants how satisfied they were with the experience. It also measured changes in self-reported pain, anxiety-related symptoms, and depressive symptoms before and after the VR session. The reported data shows that for the main measure — patient satisfaction — 9 participants reported being satisfied or very satisfied with the VR session, while 11 agreed with the statement that the VR device was helpful. For the additional measures, pain scores (rated on a 0–10 scale, where 0 means no pain and 10 means the worst possible pain) changed by an average of −0.88 points, meaning scores were slightly lower after the session. Anxiety-related symptoms, measured using a questionnaire scored from 0 to 21, showed an average change of −1.80 points. Depressive symptoms, measured on a scale from 0 to 27, showed an average change of −1.85 points. In both cases, a negative number means scores were lower after the session compared to before. It is worth noting that this was a small study with a single group — there was no comparison group receiving a different or no treatment — so the reported numbers reflect changes within the one group of 20 participants 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.

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

• NCT00586209 · results posted 2 February 2021

  According to the results reported on ClinicalTrials.gov, this trial looked at the effects of L-glutamine (an amino acid supplement) compared to a placebo (an inactive treatment) in people with sickle cell anaemia — a inherited blood condition. A total of 15 people took part: 5 received L-glutamine and 10 received the placebo. All 15 participants completed the study. The trial was measuring things like how long participants could exercise, breathing patterns during exercise, how often they experienced painful crises, how much pain relief medication they needed, and their overall level of ongoing pain. The reported data shows that, unfortunately, no actual numbers or results were submitted to ClinicalTrials.gov for any of the outcome measures — neither the primary measures (exercise endurance and breathing during exercise) nor the secondary measures (painful crises, pain medication use, and chronic pain levels). This means the specific figures for these outcomes were not reported, and it is not possible to describe what the measurements showed. Because no results data was provided for any of the measures assessed in this trial, it is not possible to draw any conclusions from the numbers. 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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• NCT00125788 · results posted 29 January 2021

  According to the results reported on ClinicalTrials.gov, this trial enrolled 70 people with sickle cell disease — 37 in the investigational product group (receiving oral L-glutamine, an amino acid supplement) and 33 in the placebo group (receiving an inactive substance). The trial ran for 48 weeks and was primarily measuring how often participants experienced painful sickle cell crises — episodes of severe pain that are a hallmark of the condition. A number of secondary measures were also tracked, including hospital admissions, emergency department visits, and certain blood test results. The reported data shows that, on average over the 48-week period, people in the investigational product group had 4.5 painful crises, compared to 10.8 in the placebo group. For hospital admissions related to sickle cell pain, the reported averages were 1.5 (investigational product) versus 2.3 (placebo). For emergency department visits, the reported averages were 3.7 (investigational product) versus 9.4 (placebo). Regarding the blood tests, the reported data shows that starting haemoglobin levels (a measure of red blood cell health) were similar between groups — around 8.96 g/dL for the investigational product group and 9.05 g/dL for the placebo group — with small changes recorded at later time points in both groups. Similar patterns of small changes from baseline were reported for hematocrit (the proportion of red blood cells in the blood) and reticulocyte count (a measure of young red blood cells being produced). 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 ↗

• NCT00445978 · results posted 28 January 2021

  According to the results reported on ClinicalTrials.gov, this trial enrolled 32 adults who all received the study drug, sapropterin dihydrochloride. The trial was set up in four back-to-back stages, with participants receiving increasing doses of the drug over 16 weeks (starting at 2.5 mg/kg/day and stepping up to 5, 10, and finally 20 mg/kg/day). Because there was only one group — everyone received the drug — there was no comparison group. The trial was measuring unwanted health events that appeared or worsened after starting the drug, as well as several body measurements related to blood vessel function, kidney function, and a marker of cell stress. The reported data shows that the main (primary) thing being tracked was how many participants experienced new or worsening health problems during treatment. At the lowest dose stage, 27 out of 32 participants had at least one such event recorded; at the second dose stage, 18 out of 29; at the third, 20 out of 25; and at the highest dose, 15 out of 24. The reported data also shows changes in several secondary measurements from the start of the trial to later time points. For blood vessel response (PAT score), the starting average was 1.58, with changes of +0.13, +0.29, +0.37, and +0.36 at each dose stage. For the urine cell-stress marker (8-isoprostane), the starting average was 1019.68 pg/mg, with changes of −27.58, −69.75, +24.71, and −275.19 at each stage. For the kidney-related urine measure (albumin-to-creatinine ratio), the baseline was 17.88 mg/mmol, with changes of +1.66, −5.51, −10.76, and −9.32. For a heart-related blood flow measurement (tricuspid regurgitant velocity), the baseline was 2.09 m/sec, with changes of +0.09, +0.03, +0.01, and +0.16 across the dose 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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• NCT01966731 · results posted 9 November 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT01966731) enrolled 635 participants, all of whom were in a single group receiving the medication hydroxyurea. Of those, 606 went on to start hydroxyurea treatment, and 600 completed three months of treatment. The trial was set up to measure whether a fixed dose of hydroxyurea caused harmful side effects (specifically blood-related toxic reactions), and also to look at certain blood test values and how well families were able to stick to the monthly clinic visit schedule. The reported data shows that for the primary outcome — the rate of dose-limiting toxic events (serious side effects that would suggest the dose was too high) — 5.1% of participants experienced this type of reaction. To put that in context, the trial's own pre-set thresholds were that a rate up to 20% was expected and a rate of 30% or more would raise concerns about safety. For the two secondary outcomes — changes in blood test measurements (such as fetal haemoglobin levels) and how well families managed to attend monthly clinic visits and take the medication as directed — no numerical results were reported in the data submitted to ClinicalTrials.gov. It is also worth noting that the data shows zero participants were recorded as having formally "completed" the study under the trial's own definition, though 600 did finish three months of treatment; the reasons for this discrepancy were 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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• NCT02530242 · results posted 19 October 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT02530242) involved 32 people in total, split into two groups of 16. One group was made up of "subjects" and the other of "controls" (a comparison group). The trial was measuring levels of a gas called end-tidal carbon monoxide (ETCO) — this is the amount of carbon monoxide that can be detected in a person's exhaled breath, measured in parts per million (ppm). All 16 subjects completed the study, as did 15 of the 16 controls (one person in the control group did not finish). The reported data shows that the average exhaled carbon monoxide reading in the subjects group was 4.35 ppm, compared to 0.80 ppm in the controls group. These numbers represent what was measured in each group's breath samples. No other outcome measures were reported in the submitted data beyond this single primary measure. Secondary outcome data was not reported, so no further figures are available to describe. 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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• NCT00115336 · results posted 8 October 2020

  According to the results reported on ClinicalTrials.gov, this trial enrolled 10 children in total — 6 in one group and 4 in the other. The trial was comparing two pain relief approaches for children admitted to hospital: one group received a drip (intravenous) form of a pain medicine called ketorolac along with a dummy oral tablet (placebo), while the other group received a dummy drip along with ibuprofen tablets by mouth. The main thing the trial was measuring was how long it took for each child's pain score to drop by half, using a recognised children's pain rating scale called the Oucher scale (scored 0–10, where higher numbers mean more pain). Nine of the 10 children completed the trial. The reported data shows that, on average, children in the intravenous ketorolac group reached that 50% pain reduction mark at around 58 hours, compared to around 68 hours in the oral ibuprofen group. For the additional things measured, the reported data shows the average hospital stay was about 81 hours for the ketorolac group and about 83 hours for the ibuprofen group. The total amount of strong painkiller (opioid, measured in morphine-equivalent milligrams) given by injection or drip during the study was reported as approximately 225 mg for the ketorolac group and approximately 265 mg for the ibuprofen group. The reported data also shows that no participants in either group developed signs of kidney stress (abnormal kidney blood test results) or fluid build-up in the body. Four participants in the ketorolac group and one in the ibuprofen group were reported to have blood detected in their urine on testing. It is worth noting that with only 10 participants across both groups, this was a very small trial, and the reported figures 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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• NCT03492099 · results posted 1 October 2020

  According to the results reported on ClinicalTrials.gov, this trial enrolled 47 adults in a single group called the "Buprenorphine Arm." Four participants did not complete the study, leaving 43 who finished. The trial was looking at what happened when people who were already taking strong opioid pain medicines (called "full agonist opioids") were switched over to a medicine called buprenorphine. Researchers were particularly interested in whether participants needed to go to hospital in the first 72 hours after the switch, and how certain other measures changed over the following six months. The reported data shows that 1 out of 47 participants needed to be hospitalised within 72 hours of being switched to buprenorphine — this was the main thing the trial was set up to measure. For one of the secondary measures, the reported average number of urgent care visits (to an emergency department or a specialised sickle cell clinic) was 11.86 per person in the six months before the switch, compared with 3.35 per person in the six months after. Another secondary measure looked at withdrawal symptoms using a standard 11-point checklist called the COWS score — on that scale, a score of 5–12 means mild withdrawal and 13–24 means moderate. The reported average score at the time of the switch was 8.92 (mild range), dropping to 3.32 (no withdrawal range) by the following day. Finally, the reported data shows that 38 out of the participants were still continuing buprenorphine therapy six months after starting it. 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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• NCT01479439 · results posted 29 September 2020

  According to the results reported on ClinicalTrials.gov, this trial enrolled 36 people in total across three groups based on their kidney protein levels: 15 in the "no albuminuria" group (no detectable protein leakage into urine), 13 in the "microalbuminuria" group (small amounts of protein in urine), and 8 in the "macroalbuminuria" group (larger amounts of protein in urine). All participants received the blood pressure medication losartan. The trial was measuring changes in a urine test called the albumin-to-creatinine ratio (UACR) — a way of checking how much of a protein called albumin is leaking into the urine, which can indicate how the kidneys are functioning. By the end of the study, 32 of the 36 participants had completed the trial. The reported data shows that the main thing being measured was how many people in each group had a reduction of 25% or more in their UACR after six months. According to the results reported on ClinicalTrials.gov, 7 out of 12 completers in the microalbuminuria group and 5 out of 6 completers in the macroalbuminuria group met this threshold, compared to just 1 out of 14 in the no-albuminuria group. The trial had set out to test whether at least 30% of the microalbuminuria group would meet this threshold. For the secondary measures, the reported data shows that UACR changed by a fold-change of 0.08 in the no-albuminuria group, −0.46 in the microalbuminuria group, and −0.74 in the macroalbuminuria group (a negative number here means the ratio went down from where it started). A separate measure of how well the kidneys filter the blood (creatinine clearance) showed fold-changes of 0.06, 0.12, and 0.05 across the three groups respectively, meaning all three groups showed a small increase from their starting 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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• NCT01783990 · results posted 20 August 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT01783990) enrolled a total of 150 children — 130 in the "Active" group and 20 in the "Passive" group. Of those, 125 and 18 respectively completed the study. The trial was looking at how a medicine called hydroxyurea (compared to a dummy pill, or placebo) affected the spleen in children with sickle cell disease. The spleen is an organ that helps fight infection, and sickle cell disease can damage it over time. The trial measured spleen function in three ways: a quality rating (normal, decreased, or absent), a count of abnormal red blood cells called "pitted cells" (a marker of how well the spleen is filtering the blood), and a count of particles called Howell-Jolly Bodies inside red blood cells (another marker of spleen activity). The reported data shows the following numbers at the measurement point when children turned 10 years old. For spleen function rated as "worse" compared to the start of the trial, 26 out of the hydroxyurea group and 28 out of the placebo group were recorded as worse, while 32 and 22 respectively were recorded as "not worse." When children were grouped by whether they were actually taking hydroxyurea at the time of the visit, 43 on hydroxyurea and 9 off hydroxyurea were "worse," while 45 on hydroxyurea and 8 off hydroxyurea were "not worse." For pitted cell percentage, the reported change from the starting point was −1.60 for the hydroxyurea group and −1.75 for the placebo group; among those on versus off hydroxyurea at the visit, the change was −1.80 versus −0.80. For Howell-Jolly Body counts, the reported change was 2,719.88 for the hydroxyurea group and 2,248.55 for the placebo group; among those on versus off hydroxyurea at the visit, it was 2,645.43 versus 1,833.73. 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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• NCT00890396 · results posted 20 August 2020

  According to the results reported on ClinicalTrials.gov, this trial enrolled 163 children in total — 127 in the "Active" group and 36 in the "Passive" group. Most children completed the study (122 and 34 respectively). The trial was measuring how spleen function changed over time in children with sickle cell disease who had previously taken part in a randomised controlled trial comparing the medicine hydroxyurea to a placebo (an inactive dummy treatment). Specifically, the study tracked two things: a qualitative (descriptive) rating of spleen function — whether it got worse or stayed the same/improved — and the percentage of "pitted cells" (a type of red blood cell count used as a marker of how well the spleen is working). The reported data shows that, when looking at spleen function ratings among children originally randomised to hydroxyurea, 16 out of 60 participants were recorded as having worse spleen function, compared with 15 out of 49 in the placebo group. Among children who were known to be actively taking hydroxyurea at the time of their study visit, 26 out of 88 were recorded as having worse spleen function, compared with 5 out of 18 who were off hydroxyurea at the time. For the pitted cell percentage measurements, the reported data shows small changes from baseline across all groups. Children originally randomised to hydroxyurea showed changes of around −1.55 to −1.70 percentage points, while those originally on placebo showed changes of around −1.00 to −1.40 percentage points. Among children on hydroxyurea at the time of their visit, the reported change was around −1.30 to −1.40 percentage points, compared with −0.60 to −1.70 percentage points for those off hydroxyurea at the time of their visit. 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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• NCT00006400 · results posted 19 August 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT00006400) enrolled 193 children with sickle cell disease — 96 received the medicine hydroxyurea and 97 received a placebo (a dummy treatment with no active ingredient). The trial ran for two years and was primarily measuring whether spleen function changed over that time, comparing the two groups. The spleen was assessed using a special scan at the start of the trial and again at the two-year mark, and each scan result was categorised as normal, reduced, or not working. The reported data shows that, among those who had both scans completed, 19 children in the hydroxyurea group experienced a worsening in spleen function, compared with 28 children in the placebo group. Meanwhile, 51 children in the hydroxyurea group did not show a worsening, compared with 46 in the placebo group. The trial also measured kidney function in several ways. One kidney measurement (using a test called DTPA GFR) was stopped early because the chance of finding a meaningful difference between groups was considered extremely small, and there was a small concern about radiation exposure from the test — so those figures are based on incomplete data. For the remaining kidney measures, the reported data shows changes from the starting point to two years: using one calculation method (the standard Schwartz formula), kidney function appeared to change by about +28.65 units in the hydroxyurea group and +33.36 units in the placebo group; using a newer calculation method, the change was about +10.57 units in the hydroxyurea group and +14.33 units in the placebo group. These figures represent changes in how well the kidneys were filtering blood, measured in standard units (millilitres per minute per 1.73 square metres of body surface area). 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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• NCT03226691 · results posted 20 April 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03226691) enrolled 15 participants, all of whom were in a single group that received a medicine called plerixafor. All 15 participants completed the study with none dropping out. The trial was looking at whether plerixafor could be used to move blood stem cells — the building blocks used in stem cell transplants — from the bone marrow into the bloodstream so they could be collected. Specifically, it was measuring whether enough of these stem cells could be gathered (reaching a set target amount) without any serious unwanted medical events occurring. The reported data shows that out of the 15 participants, 14 reached the target collection amount of blood stem cells without experiencing a serious adverse event (an unexpected or harmful medical occurrence considered serious in nature). The results data submitted to ClinicalTrials.gov only included this single primary outcome measure; no secondary outcome measure data was reported in the structured results, so those figures are not available to describe 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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• NCT02640573 · results posted 27 January 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT02640573) enrolled only 1 participant, who was given a treatment called hydroxyurea (also spelled hydroxurea). The trial was set up as a single-arm study — meaning there was no comparison group — and it was designed to measure changes in a type of haemoglobin called foetal haemoglobin (HbF) in response to the treatment. HbF is a form of haemoglobin (the protein in red blood cells that carries oxygen) that is sometimes measured in certain blood conditions. The reported data shows that the 1 participant who started the trial did not complete it. Because of this, no outcome measurement results were recorded or submitted for the primary outcome measure — that is, the change in HbF levels over time. The data was not reported for this outcome, so there are no numbers available to describe. In summary, this trial was not able to generate any findings about its main research question, as only one person enrolled and that person did not finish 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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• NCT00761085 · results posted 22 January 2020

  According to the results reported on ClinicalTrials.gov, this trial enrolled 47 people in total — 12 children and 11 adults who received methadone, and 12 children and 12 adults who received morphine. All participants had sickle cell disease and were experiencing a pain crisis (called a vaso-occlusive episode). The trial was primarily looking at how methadone moves through the body (its "pharmacokinetics" — meaning how it is absorbed, distributed, and cleared) in both children and adults. A secondary goal was to record participants' pain levels using a standard 0–10 scale, where 0 means no pain and 10 means the worst pain imaginable. The reported data shows that the primary measurement — a figure called AUC (area under the curve, a way of capturing how much of the drug was present in the blood over time) — was 523 units (hr\*ng/ml) for children who received methadone and 637 units for adults who received methadone. The AUC figures for the morphine groups were reported as zero, which likely reflects that this measurement was only applicable to the methadone groups. For the secondary pain scale measure, the reported data shows average scores of 8 out of 10 for children on methadone, 4 out of 10 for children on morphine, 5 out of 10 for adults on methadone, and 5 out of 10 for adults on morphine. It is worth noting that not all participants completed the study — six children and four adults in the morphine groups did not finish, though the reasons were not detailed 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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• NCT02013375 · results posted 28 August 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 2 participants, all of whom were in a single group receiving a haploidentical stem cell transplant — a type of transplant that uses a donor who is only a partial genetic match, such as a parent or sibling. The trial was studying this transplant approach in people with sickle cell disease, using a combination of medicines to prepare the body (called conditioning) along with a low dose of whole-body radiation. Neither of the 2 participants was recorded as having completed the study. The reported data shows that the main thing being measured was whether the transplanted cells successfully "engrafted" — that is, took hold and started working in the recipient's body — by day 60 after the transplant. The result reported for this measure was 0 out of 2 participants. The trial also planned to track complications of sickle cell disease, survival, and rates of a condition called graft-versus-host disease (where transplanted cells can attack the recipient's body), as well as infections and transplant-related deaths. However, the reported data shows that no numerical results were submitted to ClinicalTrials.gov for any of these secondary measures. It is worth noting that with only 2 participants enrolled and neither completing the study, the amount of information available from this trial 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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• NCT02098993 · results posted 16 July 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02098993) enrolled a total of 7 people — 4 in a group receiving unfractionated heparin (a blood-thinning medication) and 3 in a group receiving standard of care. All 7 participants completed the study. The trial was measuring how long participants stayed in hospital, as well as tracking several other things over time, including low blood oxygen levels, fever, elevated white blood cell counts (a marker the body is responding to something), pain scores, and the total amount of opioid pain medication given. The reported data shows that, for the primary outcome, the unfractionated heparin group spent an average of around 279 hours in hospital, compared with around 127 hours for the standard of care group. For the secondary outcomes, the reported data shows the heparin group experienced low blood oxygen levels for an average of about 118 hours versus about 51 hours in the standard care group; fever lasted an average of about 25 hours in the heparin group and 0 hours in the standard care group; and elevated white blood cell counts lasted about 118 hours versus about 53 hours respectively. For pain, the heparin group reported moderate-to-severe pain for an average of about 89 hours, while the standard care group reported about 118 hours. The reported total opioid doses averaged around 3,447 mg per person in the heparin group and around 1,167 mg per person in the standard care group. It is worth noting that with only 7 participants across both groups, this was a very small study, and the reported figures should be understood in that context. The reported data shows numbers for each group, but no further statistical analysis results (such as measures of certainty or significance) were included in the data 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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• NCT02504619 · results posted 26 June 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02504619) enrolled just one participant, who received a stem cell product called CordIn. The trial was measuring three things: whether the infusion caused any serious side effects within the first 24 hours, whether the participant's body accepted donor cells within 42 days of the transplant, and whether the participant was still alive one year after the transplant. The reported data shows that the single participant completed the study without dropping out. On the primary outcome of infusion-related reactions in the first 24 hours, the result was reported as 1 participant — meaning the one enrolled person was assessed for this measure. For the second primary outcome, the reported data shows that the participant had donor-derived cells present at 42 days after the transplant, which the trial description notes represents 100% of those enrolled. For the secondary outcome of survival at 365 days, the reported data shows that 1 participant (again, the only person enrolled) was alive at the one-year mark. It is important to note that because only one person took part, these numbers reflect the experience of a single individual and cannot be used to draw broader conclusions. The data does not include detailed breakdowns of side effect severity grades, which the trial planned to report — that information was not provided 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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• NCT01572922 · results posted 21 May 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT01572922) enrolled 142 participants, with 139 completing the study and 3 not completing it. There was one group of participants who had too much iron stored in their liver (referred to as "iron-overloaded patients"). The trial was investigating whether a type of MRI scan could be used to measure how much iron is in the liver — potentially as an alternative to a liver biopsy, which involves taking a small sample of liver tissue with a needle. The reported data shows that when liver iron was measured directly through biopsy, the average result was 19.8 micrograms (mcg) of iron. The MRI measurements work by tracking how quickly a signal fades during scanning — a faster fade suggests more iron is present, and this is captured as a number called R2* (measured in hertz, or Hz). The reported data shows that using one MRI method (called UTE, a specific scanning technique), the average R2* reading was 864.4 Hz for the primary outcome, and 319.2 Hz in a secondary measurement using the same technique. A second MRI method (called GRE, another scanning approach) gave an average R2* of 333.8 Hz. For participants who also had blood iron tests, the average R2* using the UTE method was 340.8 Hz, and the average blood iron level in that group was 156.5 micrograms per decilitre (ug/dL). 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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• NCT00895154 · results posted 15 May 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 18 children across two groups: 8 in a General Tutoring group and 10 in a Tutoring plus Memory Training group. By the end of the study, 7 and 8 participants respectively had completed the trial (1 and 2 did not complete it). The trial was measuring scores on standardised tests of intelligence and academic skills — specifically, IQ scores and scores for word reading, reading fluency, and maths calculations — to compare how the two groups performed after their respective programmes. The reported data shows that on the IQ tests (where 100 is the average score for the general population), the Full Scale IQ scores at the end of the study were 86.4 for the General Tutoring group and 88.0 for the Tutoring plus Memory Training group. Verbal IQ scores were 87.9 and 87.8 respectively, and Performance IQ (the more hands-on, visual reasoning portion) scores were 87.3 and 90.4. For the academic skills tests (where 50 is the average score), word reading scores were 42.1 and 44.0; reading fluency scores were 40.1 and 40.4; and maths calculation scores were 36.9 and 44.6, for the General Tutoring and Tutoring plus Memory Training groups respectively. The reported data does not include baseline scores or statistical comparisons between the groups, so the figures above reflect end-of-study scores 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.

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

• NCT02697240 · results posted 17 April 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 4 participants, all of whom completed the study. There was only one treatment group, and all participants received intravenous citrulline (a naturally occurring amino acid given through a drip). The trial was measuring two main things: whether participants experienced any unwanted side effects, and how much citrulline was detectable in the blood after the treatment was given. The reported data shows that all 4 participants experienced at least one adverse event (an unwanted or unexpected medical occurrence noted during the trial). The trial also measured the level of citrulline in the blood, which was reported as 5 micromol/hr/kg — this is a unit describing the amount of the substance circulating in the bloodstream over time, adjusted for body weight. No further breakdown of the adverse events or additional outcome details appear to have been submitted to ClinicalTrials.gov, so more specific information on those points cannot be reported here. It is worth noting that with only 4 participants, this was a very small study — likely an early-phase trial designed to gather initial information rather than draw broad conclusions. The reported numbers reflect only what was observed in this small group and 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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• NCT01954927 · results posted 2 April 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT01954927) enrolled 90 children in total — 45 in a group that received gabapentin (a medicine sometimes used for pain) and 45 in a group that received a placebo (a dummy treatment with no active ingredient). The trial was looking at pain during a sickle cell disease episode called a vaso-occlusive crisis, where blocked blood flow causes severe pain. The main thing being measured was whether a child's pain score dropped by at least one-third (33%) within three hours of taking the study drug — this was called a "successful intervention." The reported data shows that, for the primary outcome measured at three hours after taking the study drug, 13 out of 45 children in the gabapentin group and 17 out of 45 in the placebo group were recorded as having a successful pain reduction (the remaining 27 and 25 respectively did not meet that threshold). For a secondary measure — the total amount of morphine-equivalent pain relief medicine given during those three hours — the reported data shows an average of 0.12 mg per kilogram of body weight in the gabapentin group and 0.13 mg per kilogram in the placebo group. A similar pattern was seen at a later time point (when doctors decided whether to admit or send the child home): 10 in the gabapentin group and 17 in the placebo group had a successful pain reduction. Hospital admission numbers were also similar between groups — 32 admitted in each group, and 10 (gabapentin) versus 12 (placebo) discharged home. The reported change in pain score from the time of taking the study drug to three hours later was 0 points in the gabapentin group and 0.5 points in the placebo group, on a scale where 0 means no pain and 10 means the worst possible pain. 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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• NCT01960413 · results posted 26 March 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 46 people with sickle cell disease — 24 in one group and 22 in another. All participants were already taking a medicine called hydroxyurea, and the trial then added either montelukast (a tablet more commonly known as a preventer for asthma and allergies) or a placebo (a dummy pill with no active ingredient) to see what difference, if any, this made. The trial ran until 20 people in the montelukast group and 21 in the placebo group completed it. The main thing being measured was the change in the blood level of a protein called sVCAM (soluble Vascular Cell Adhesion Molecule-1). This protein is thought to be involved in the way blood cells stick to blood vessel walls. The reported data shows that, on average, the level of this protein went **down by 3.2 ng/mL** (nanograms per millilitre, a very small unit of measurement) in the group that received montelukast added to hydroxyurea. In the group that received the placebo added to hydroxyurea, the level went **up by 4.17 ng/mL**. The trial had originally been designed around detecting a much larger change — around 106 ng/mL — so the reported numbers are notably smaller than the target the study was set up to find. No other outcome measures were included in the submitted results 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.

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

• NCT02061202 · results posted 11 March 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 54 people in total — 36 received a medicine called mometasone furoate (an inhaled corticosteroid, a type of anti-inflammatory medicine breathed in through an inhaler) and 18 received a placebo (a dummy treatment with no active ingredient). The trial was exploring whether it was practical and feasible to run a full study of this inhaled medicine in people with sickle cell disease, and it tracked a range of measurements including pain, lung inflammation, a marker of blood vessel health, quality of life, and how well participants stuck to their medication. The reported data shows that 35 out of 36 participants in the mometasone furoate group and 17 out of 18 in the placebo group completed the follow-up period. For the secondary measurements, the mometasone furoate group showed a change of 0.63 ppb (parts per billion) in exhaled nitric oxide (a breath test used to indicate lung inflammation), compared with 2.71 ppb in the placebo group. A blood vessel health marker called sVCAM changed by −182.47 ng/mL in the mometasone furoate group and +170.25 ng/mL in the placebo group. On a quality-of-life pain score (where a higher number meant more impact from pain), the mometasone furoate group changed by 2.8 points and the placebo group by 6.9 points. A self-reported pain score (0–10, where higher means more pain) changed by 2.09 in the mometasone furoate group and 2.82 in the placebo group. Medication adherence scores (0–25, where higher means better adherence) were 17.7 and 17.1 respectively. No information about why these numbers differed, or whether the differences were meaningful, was reported in the structured 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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• NCT02114515 · results posted 15 February 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT02114515) involved 1,029 people in total — 511 in a "Usual Care" group and 518 in a "Usual Care + PArTNER" group. PArTNER appears to have been a program added on top of standard hospital care. The trial was measuring changes in participants' anxiety levels and their sense of feeling supported (both with information and emotionally) from when they entered the study to 30 days after leaving hospital. Around 370–380 people in each group completed the study. The reported data shows the following for the two main (primary) outcomes, measured using a standardised scoring system called a "T-score" (a number used to compare people against a general population average). For anxiety, a lower score after 30 days means less anxiety: the Usual Care group's score changed by −0.2 (very little change), while the Usual Care + PArTNER group's score changed by −1.7 (a slightly larger reduction). For informational support — that is, how well people felt they received useful information — a higher score means more support felt: the Usual Care group's score rose by 2.3 points and the Usual Care + PArTNER group's score rose by 2.5 points. For the secondary outcomes, the reported data shows very similar small changes between the two groups across emotional support (both −0.1), practical/hands-on support (Usual Care +1.0, PArTNER +0.6), physical health (Usual Care +4.6, PArTNER +4.4), and mental health (Usual Care +0.8, PArTNER +0.6). 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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• NCT03291613 · results posted 6 February 2019

  According to the results reported on ClinicalTrials.gov, this trial involved 13 participants who tested an app called Pinpoint. The study was measuring how easy the app was to use. Twelve of the 13 participants completed the trial, and one did not finish. The main thing being measured was a standard usability questionnaire made up of ten questions, where participants rated how user-friendly the app felt. The reported data shows that the average score across all participants was 86.67 out of 100. According to the questionnaire's own scale, a score of 68 or above is considered above average for usability — the reported average score of 86.67 sat above that 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.

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• NCT00749515 · results posted 6 February 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 15 people in total — 10 who had previously shown a poor response to deferasirox (a medicine used to remove excess iron from the body) and 5 who had shown a good response. All 15 participants completed the study. The trial was measuring how the body processed deferasirox — specifically, how much of the drug was present in the blood over time, how quickly it was cleared from the body, and how it was distributed through the body's tissues. Participants also underwent a separate infusion of another iron-removing medicine (deferoxamine) to assess their iron levels. The study also looked at genetic differences between the two groups that might help explain any differences in how the drug was handled by the body. The reported data shows several differences between the two groups in how deferasirox moved through their bodies. The total amount of drug detected in the blood over 24 hours (called "area under the curve") was reported as 479.59 micromole/litre\*hour in the poor-response group, compared with 1,123.11 in the good-response group — meaning the poor-response group had considerably less drug circulating in their blood. The time it took for the drug level to fall by half ("half-life") was reported as 6.08 hours in the poor-response group versus 7.83 hours in the good-response group. The rate at which the body cleared the drug was reported as 1.30 litres/hour in the poor-response group compared with 0.61 litres/hour in the good-response group, suggesting the poor-response group cleared the drug more quickly. On the genetic side, all 15 participants were tested for variations in four genes thought to be involved in how the body processes deferasirox, though a breakdown of how many in each group carried specific variations was 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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• NCT02731157 · results posted 25 January 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 4 participants in total. It was a small study that compared two types of red blood cell (RBC) transfusions in people with sickle cell disease who receive regular exchange transfusions — a procedure where some of the patient's blood is replaced with donor blood. The two types compared were "standard" red blood cells (stored in the usual way) and "rejuvenated" red blood cells (treated with a solution intended to restore some properties that stored blood can lose over time). Each participant went through multiple rounds of both types of transfusion. The main thing being measured was how quickly the donor red blood cells were replaced by the patient's own sickle-cell red blood cells after each transfusion — tracked by measuring a specific type of haemoglobin (the oxygen-carrying protein in red blood cells) called HbA. The reported data shows that for the primary outcome — the average daily percentage decrease in donor HbA after transfusion — the figure was 0.629% per day for the rejuvenated red blood cell transfusions, and 0.659% per day for the standard red blood cell transfusions. In plain terms, this measures roughly how fast the transfused donor blood "fades" from the bloodstream each day. The reported data shows that several secondary outcomes were also planned — including measures of red blood cell particles, clotting-related activity, and oxygen-carrying capacity — however, no numerical results for any of these secondary outcomes were reported in the submitted data on ClinicalTrials.gov. It is worth noting that only 4 people took part, which is a very small number, and one participant did not complete all stages of the trial. Because of the small size, these figures should be understood only as preliminary reported measurements from this specific 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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• NCT03041909 · results posted 2 January 2019

  According to the results reported on ClinicalTrials.gov, this trial looked at a medicine called GBT440 and involved a total of 5 participants. These participants were divided into three groups based on how long they took the medicine — one group of 3 people took it for 2 months, one person took it for 4 months, and one person took it for 6 months. All 5 participants who started the trial completed it. The trial was primarily measuring how many participants experienced unwanted side effects (called "treatment-emergent adverse events") while taking GBT440. It also aimed to measure changes in a blood protein called haemoglobin (which carries oxygen around the body), how the body processed the medicine in the blood, and markers of red blood cell breakdown. The reported data shows that, when it came to the primary measure — unwanted side effects during the dosing period — 2 out of 3 participants in the 2-month group, 1 out of 1 in the 4-month group, and 1 out of 1 in the 6-month group each experienced at least one such event. For all three secondary outcome measures — haemoglobin levels, how the medicine was absorbed into the blood, and markers of red blood cell breakdown — the data was not reported, so no numbers are available for those 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.

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

• NCT01976416 · results posted 1 October 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT01976416) enrolled 208 children in total — 104 who received hydroxyurea and 104 who received a placebo (a dummy treatment with no active ingredient). Nearly all participants finished the trial, with 102 completing in each group and only 2 dropping out from each side. The trial was measuring how often children in each group experienced episodes of malaria — defined as having a malaria parasite detected on a blood test when a child was brought in with a fever. The reported data shows that the hydroxyurea group had 5 malaria episodes per 100 patient-years, while the placebo group had 7 malaria episodes per 100 patient-years. "Patient-years" is simply a way of accounting for the total amount of time all participants were followed — so these figures represent the rate at which malaria episodes occurred across the whole group over time. No other outcome measures were 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.

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

• NCT02200510 · results posted 15 June 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT02200510) involved people living with sickle cell disease and compared two groups: a Self-Management Group (31 participants at the start) and a Patient Portal group (47 participants at the start). The trial was measuring whether participants' confidence in managing their own sickle cell disease changed over a 6-week period. Of those who started, 22 people in the Self-Management Group and 44 people in the Patient Portal group completed the study. The reported data shows that the main thing being measured was a change in scores on the Sickle Cell Self-Efficacy Scale (SCSES) — a questionnaire designed to capture how confident someone feels in managing their sickle cell disease. The scale runs from 9 (least confident) to 45 (most confident). According to the results reported on ClinicalTrials.gov, at the 6-week point the Self-Management Group had an average score of 33.2, and the Patient Portal group had an average score of 33.09. It is worth noting that the data as submitted does not appear to include the participants' starting (baseline) scores separately, so a clear before-and-after comparison figure was not reported in the structured 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.

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

• NCT02214121 · results posted 11 May 2018

  According to the results reported on ClinicalTrials.gov, this trial involved a total of 71 participants across two parts. Part A enrolled 46 people, all of whom received the study medicine, ticagrelor, at various doses. Part B enrolled 25 people, with 17 receiving ticagrelor and 8 receiving a placebo (a dummy treatment with no active ingredient). The trial was measuring how the medicine moved through the body at different doses, and how it affected a specific marker in the blood called P2Y12 Reaction Units (PRU) — a measure of how active certain blood cells called platelets are. Higher PRU numbers generally indicate more active platelets. The reported data shows that in Part A, PRU readings across the different ticagrelor dose groups ranged from around 205 to 320 units at one time point, and from roughly 138 to 295 units at another. In Part B, the reported PRU readings were around 283 for the ticagrelor group and 313 for the placebo group at one time point, and around 260 and 217 respectively at another. The trial also tracked how much of the medicine entered the bloodstream. In Part A, the peak blood concentration of ticagrelor (the highest level measured in the blood) ranged from about 14 ng/mL at the lowest dose up to about 567 ng/mL at the highest dose, with the total amount of medicine the body was exposed to over time rising correspondingly with each higher dose. In Part B, only one ticagrelor dose group was reported, with a peak blood level of about 16 ng/mL. Placebo concentration figures were not reported for Part B, which would be expected as the placebo contains no active medicine. 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 ↗

• NCT01788631 · results posted 7 February 2018

  According to the results reported on ClinicalTrials.gov, this trial looked at a drug called regadenoson in people with sickle cell anaemia who were in hospital with pain or a complication called acute chest syndrome. A total of 100 people were randomly assigned to receive either regadenoson (53 people) or a dummy treatment called a placebo (47 people). The trial's main question was whether regadenoson could reduce the activity of a particular type of immune cell — called an iNKT cell — by 70% or more compared to the placebo group. The reported data shows that, for the main measure, 21 out of the regadenoson group and 10 out of the placebo group had that level of reduction in iNKT cell activity. For the secondary measures, the reported data shows that average hospital stay was nearly identical between the two groups — about 3.96 days for regadenoson and 3.99 days for placebo. When it came to breathing-related symptoms improving, 10 participants in the regadenoson group and 9 in the placebo group met the criteria for improvement. Pain relief medication (opioid) use was reported as 0.03 mg/kg/hr in the regadenoson group and 0.04 mg/kg/hr in the placebo group. Two markers of inflammation in the body were also measured: one (called A2A) was reported at about 86% of the starting level in the regadenoson group and about 101% in the placebo group; another (called IL-4) was reported at about 86% of the starting level in the regadenoson group and about 101% in the placebo group. No further breakdown of the results — such as whether the differences between groups were considered meaningful by the researchers — was included in the data submitted to ClinicalTrials.gov, so those details cannot be 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.

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

• NCT02114203 · results posted 14 December 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT02114203) looked at a drug called PF-04447943, tested in people with sickle cell disease. A total of 30 participants took part: 7 received a lower dose (5 mg twice daily), 16 received a higher dose (25 mg twice daily), and 7 received a placebo (a dummy treatment with no active ingredient). The trial was primarily measuring safety-related changes — things like blood pressure, heart rhythm (using ECG recordings), neurological function, physical examination findings, sickle cell disease symptoms, and whether participants experienced any unwanted medical events during the study. The reported data shows that no participants in any group had notable changes in neurological function, physical examination findings, or sickle cell disease symptoms that the investigators considered potentially clinically important. For blood pressure, small numbers of participants had large changes — for example, 2 people in the lower-dose group and 1 in the placebo group had a significant drop in diastolic blood pressure (the lower number in a blood pressure reading). For heart rhythm, 3 participants in the higher-dose group and 2 in the placebo group had a QTcF interval (a specific measure of the heart's electrical activity) fall into a particular monitoring range. Regarding unwanted medical events, all 7 participants in the lower-dose group and 13 of 16 in the higher-dose group, as well as all 7 placebo participants, experienced at least one adverse event. Serious adverse events were reported in 2 participants in the lower-dose group and 1 in the higher-dose group, with none in the placebo group. One participant in the higher-dose group discontinued due to an adverse event; no participants in the other groups did. 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 ↗

• NCT02482298 · results posted 14 December 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 87 people with sickle cell disease across three groups: 30 received a placebo (dummy treatment) twice daily, 27 received a lower dose of ticagrelor (10 mg twice daily), and 30 received a higher dose of ticagrelor (45 mg twice daily). The trial was measuring whether ticagrelor, a medicine that affects how blood platelets clump together, made any difference to the number of days participants experienced pain, the intensity of that pain, and how often they needed pain-relief medicines. By the end of the study, 28, 24, and 27 participants in each group respectively had completed the trial. The reported data shows that the main thing being measured was the change in the proportion of days participants recorded pain in a daily electronic diary. All three groups showed a reduction from their starting point: the placebo group's proportion of pain days fell by about 0.18, the lower-dose ticagrelor group fell by about 0.14, and the higher-dose ticagrelor group fell by about 0.10. For average daily worst pain intensity — scored on a scale of 0 (no pain) to 10 (worst imaginable) — the reported average scores during the study were 1.02 for the placebo group, 1.15 for the lower-dose group, and 1.74 for the higher-dose group. The reported data also shows that the proportion of days participants used pain-relief medicines fell by about 0.20 in the placebo group, 0.08 in the lower-dose group, and 0.10 in the higher-dose group. The trial also tracked certain bleeding events. The reported data shows that 2 participants in each group experienced major or clinically relevant non-major bleeding events, with similar small numbers of events across all three groups. These figures were recorded as part of monitoring for safety-related outcomes, and no further breakdown beyond what was submitted to ClinicalTrials.gov is available 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.

  View reported results on ClinicalTrials.gov ↗

• NCT01565616 · results posted 29 September 2017

  According to the results reported on ClinicalTrials.gov, 22 people with sickle cell disease who had received a bone marrow transplant took part in this trial. All 22 completed the study. The trial was divided into two parts: one involving 5 participants who received transplants from related donors, and a second involving 17 participants who received transplants from either related or unrelated donors. The trial was measuring how many participants remained free of sickle cell disease events after their transplant, as well as tracking things like graft failure (whether the transplanted cells took hold), a condition called graft versus host disease (GVHD — where donated cells can react against the recipient's body), overall survival, and how quickly key blood cells recovered. The reported data shows that 19 out of 22 participants were classified as event-free survivors, meaning they had stable donor blood cell production with no new signs of sickle cell disease. For overall survival, 20 out of 22 participants were alive at the end of the study period. Regarding graft failure, the data shows 0 participants experienced primary graft failure (where the donated cells never established themselves) and 1 participant experienced secondary graft failure (where the donated cells initially worked but later failed). For acute GVHD, 3 participants experienced Grade II, 1 experienced Grade III, and 0 experienced Grade IV. For chronic GVHD, 3 participants had mild, 2 had moderate, and 1 had severe disease. The reported data also shows that, on average, it took 17 days for a type of infection-fighting white blood cell (neutrophils) to recover, and 21 days for platelets (cells that help blood clot) to recover. 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 ↗

• NCT01179217 · results posted 10 August 2017

  According to the results reported on ClinicalTrials.gov, this trial looked at oral L-glutamine (an amino acid supplement) as a potential treatment for sickle cell anaemia and a related condition called beta-0 thalassemia. A total of 152 people were assigned to take L-glutamine and 78 were assigned to take a placebo (an inactive powder called maltodextrin, with no active ingredient). Over 48 weeks, the trial tracked how often participants experienced sickle cell crises (episodes of severe pain caused by the disease), how often they were hospitalised for pain, how often they visited an emergency room, and also monitored basic health measurements like blood levels and blood pressure. The reported data shows that, on average, people in the L-glutamine group had 3 recorded sickle cell crises over the 48 weeks, compared with 4 in the placebo group. For hospitalisations due to sickle cell pain, the L-glutamine group recorded an average of 2 compared with 3 in the placebo group. Emergency room or medical facility visits were recorded as an average of 1 in both groups. For blood-related measurements, haemoglobin levels (a measure of red blood cell health, in grams per decilitre) started at around 8.82 in the L-glutamine group and 8.71 in the placebo group, with small changes recorded at weeks 4, 24, and 48 that were similar between the two groups. Hematocrit (the proportion of blood made up of red blood cells) and blood pressure figures also showed small and broadly comparable changes across both groups over the course of the study. It is worth noting that a notable number of participants did not complete the study — 55 out of 152 in the L-glutamine group and 19 out of 78 in the placebo group — though the reasons for this were not detailed 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.

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

• NCT02222246 · results posted 4 August 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 53 people in each of two groups — 106 participants in total. One group received a standard, fixed dose of a pain-relieving medicine (morphine sulfate or hydromorphone) in the emergency department, while the other group received a dose calculated specifically for that individual patient. The trial's main goal was to measure how much each person's self-rated pain score changed from when they arrived at the emergency department to when they left. Pain was scored on a 0–100 scale, where 0 meant no pain and 100 meant the worst imaginable pain. It is worth noting that only 26 people in each group completed the study, with 27 in each group not completing it; the data does not explain why. The reported data shows that, on average, people in the standard-dose group had a pain score reduction of 26.4 points from arrival to discharge, while people in the patient-specific-dose group had a reported reduction of 43.0 points over the same period. A secondary measure tracked pain scores every 30 minutes; both groups started with very similar scores (around 82 out of 100), and by discharge the standard-dose group's average score was reported as 55.7, compared with 40.9 for the patient-specific-dose group. Other secondary measures tracked things like nausea, vomiting, and drops in blood pressure during the visit. For nausea, the reported data shows 38 visits in the patient-specific-dose group and 16 in the standard-dose group involved at least one episode of nausea. For vomiting, 10 visits in the standard-dose group and 8 in the patient-specific-dose group involved at least one episode. For significant drops in blood pressure (20% or more below the starting level), the numbers were similarly small across both groups for both the upper (systolic) and lower (diastolic) blood pressure readings. 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 ↗

• NCT02301936 · results posted 25 May 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled a total of 10 people with hepatitis C. Nine participants were assigned to take a combination treatment (ledipasvir/sofosbuvir, referred to here as LDV/SOF) for 12 weeks, and one participant was assigned to take the same treatment for 24 weeks. The trial was primarily measuring two things: how many participants had no detectable hepatitis C virus in their blood 12 weeks after finishing treatment (known as SVR12, a standard way of checking whether the virus remains undetectable after therapy), and how many participants permanently stopped taking the study drug because of an unwanted side effect. The reported data shows that in the 12-week group, 88.9% of participants (roughly 8 out of 9) had no detectable virus at the 12-week post-treatment check. In the 24-week group, 100% of participants (the single participant) had no detectable virus at that same point. Regarding stopping treatment early due to side effects, 0% of participants in either group permanently discontinued the study drug for that reason. A secondary measure looked at "virologic failure" — meaning the virus became detectable again or never dropped low enough during or after treatment — and the reported data shows this occurred in 11.1% of the 12-week group (approximately 1 person) and 0% of the 24-week group. It is worth noting that this was a very small trial — just 10 people in total — and the reported data should be understood in that context. The secondary measures also tracked how the level of virus in the blood changed over the course of treatment, and those numbers showed reductions across both groups at various time points, though the full breakdown of those figures is complex. 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 ↗

• NCT00940901 · results posted 13 April 2017

  According to the results reported on ClinicalTrials.gov, this trial involved 13 people in total — 6 in the sildenafil group and 7 in a group that started on a placebo (an inactive pill) before switching to sildenafil. The trial was measuring whether the medication sildenafil could reduce the number of unwanted, prolonged erections (called priapic episodes) in participants. It ran in two phases of eight weeks each. By the second phase, a number of participants did not complete the study — 2 from the sildenafil group and 3 from the placebo-then-sildenafil group did not finish. The main thing the trial measured was whether participants experienced at least a 50% reduction in the number of these episodes, based on a diary log they filled in. The reported data shows that in the first phase, 3 out of 6 participants in the sildenafil group and 3 out of 7 in the placebo-then-sildenafil group met this threshold. In the second phase, the reported data shows 2 out of 6 in the sildenafil group and 3 out of 7 in the placebo-then-sildenafil group reached that same level of reduction. No secondary outcome data appears to have been reported in the structured results submitted. It is worth noting that this was a very small trial, and the results as submitted to ClinicalTrials.gov reflect only the raw numbers of participants who met the measurement threshold — no additional detail beyond those figures was 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.

  View reported results on ClinicalTrials.gov ↗

• NCT01987908 · results posted 24 March 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT01987908) looked at a study product being investigated for sickle cell disease — a condition where red blood cells are abnormally shaped and can cause serious health problems. The trial enrolled 23 participants in total. It began with a "placebo lead-in" phase where all 23 participants received a dummy treatment (placebo), after which 14 completed that stage and moved into a double-blind treatment period. In that treatment period, 11 participants received the study product and 3 received the placebo. A smaller group then continued into a follow-up observation period. The trial's primary focus was on recording and counting adverse events (unexpected or unwanted health changes) across all stages, as well as measuring how the study product moved through the body (pharmacokinetics). The reported data shows that during the double-blind treatment period, 9 out of 11 participants in the study product group and 3 out of 3 participants in the placebo group experienced at least one adverse event of any kind. Within those groups, 8 and 2 participants respectively experienced adverse events that were classified as related to sickle cell disease specifically (such as pain episodes requiring intravenous pain relief, hospitalisation, or acute chest syndrome). During the earlier placebo lead-in phase, 21 of the 23 participants reported at least one adverse event, with 12 experiencing sickle cell-related symptoms. During the post-treatment observation period, 4 out of 10 participants who had received the study product reported adverse events, compared with none of the 2 participants who had received placebo in that stage. The reported data shows that pharmacokinetic measurements (how the study product was absorbed and processed by the body) were listed as a planned outcome, but no numerical results for those measurements were included in the data submitted to ClinicalTrials.gov. It is worth noting that because this was a very small trial, the numbers of participants in each group were quite low, which means these figures should be understood as early-stage information 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.

  View reported results on ClinicalTrials.gov ↗

• NCT00427661 · results posted 18 August 2016

  According to the results reported on ClinicalTrials.gov, this trial enrolled 8 participants, all of whom had severe sickle cell disease (a serious inherited blood condition). All 8 participants completed the study — none dropped out. The trial was looking at what happened after participants received a bone marrow transplant (a procedure where blood stem cells from a donor are given to replace the patient's own), specifically focusing on two things: whether participants' bodies accepted the new cells (called "engraftment"), and whether they developed a condition called graft-versus-host disease, or GVHD (where donor cells can sometimes attack the recipient's body). The reported data shows that, within one year of the bone marrow transplant, 6 out of the 8 participants showed engraftment — meaning their bodies appeared to be carrying a mix of their own and the donor's blood cells at the one-year mark. Regarding GVHD, the reported data shows that 2 out of the 8 participants developed this condition within the first year. For a more specific measure — a more serious form called "Grade 2–4 acute GVHD" (a grading scale where higher numbers reflect greater severity) — the reported data shows that 1 out of the 8 participants was recorded as experiencing this level. It is worth noting that this was a very small study of only 8 people, and the results as submitted reflect measurements at specific points in time for this particular group 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.

  View reported results on ClinicalTrials.gov ↗

• NCT01794000 · results posted 27 July 2016

  According to the results reported on ClinicalTrials.gov, this trial (NCT01794000) enrolled 341 children and teenagers with sickle cell disease — 171 in the prasugrel group and 170 in the placebo group. The trial was measuring whether prasugrel, a medicine that reduces blood clotting, had any impact on "vaso-occlusive crises" (VOCs) — painful episodes caused by blocked blood flow that are a hallmark of sickle cell disease — as well as related outcomes like pain levels and hospital admissions. The main phase of the trial was a double-blind phase, meaning neither participants nor researchers knew who was receiving which treatment. Very few participants completed the full double-blind phase (2 in the prasugrel group and 4 in the placebo group), with the large majority not completing it. The reported data shows that for the primary outcome — the rate of VOC episodes per person per year — the prasugrel group recorded approximately 2.30 events per person per year, compared with approximately 2.77 in the placebo group. For the secondary outcomes, the reported data shows that the percentage of days in a month that participants experienced any pain was approximately 17.5% in the prasugrel group and 17.7% in the placebo group. Average daily pain scores (on a scale of 0 to 10, where 0 means no pain) were reported as 0.71 in the prasugrel group and 0.61 in the placebo group. The rate of hospitalisations for VOC was reported as approximately 1.06 per person per year (prasugrel) versus 1.13 (placebo). The rate of acute chest syndrome — a serious lung complication — was approximately 0.11 per person per year in both 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.

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

• NCT01531387 · results posted 9 December 2015

  According to the results reported on ClinicalTrials.gov, this trial (known as the SCATE trial) enrolled 22 children with sickle cell disease — 11 in a group receiving a medicine called hydroxyurea, and 11 in a group receiving standard care (observation only). The trial was designed to look at whether hydroxyurea could reduce the risk of stroke in children who already had a moderate level of stroke risk, as measured by the speed of blood flow in brain arteries using a painless ultrasound scan called transcranial doppler (TCD). It also planned to track neurological events (such as strokes), other sickle cell-related health events, and quality of life over time. The reported data shows that no outcome measure results were submitted to ClinicalTrials.gov for any of the primary or secondary outcomes. This includes the main measure — how many children's brain blood-flow speeds went from a "moderate risk" level to a "high risk" level — as well as all secondary measures, such as changes in blood-flow speeds over time, rates of stroke or other neurological events, other sickle cell complications, and quality of life scores. The data for these outcomes was not reported. It is also noted that zero participants in either group were recorded as having "completed" the study, with all 22 participants listed as "not completed," though no further explanation for this is 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.

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

• NCT01363908 · results posted 27 May 2015

  According to the results reported on ClinicalTrials.gov, this trial enrolled 30 children and teenagers in total — 14 aged 6 to under 12 years, and 16 aged 12 to under 18 years. The trial was measuring how the body processes a drug called SPD602 (taken as a capsule) in these two age groups. Specifically, the researchers tracked how the drug moved through the bloodstream and was removed from the body via urine after a single dose — this type of measurement is called pharmacokinetics, meaning it looks at how a medicine travels through the body over time. A smaller subgroup of 8 participants from each age group took part in the detailed blood-sampling phase, while the larger group participated in a longer dosing phase. The reported data shows that, after a single oral dose of SPD602, the peak level of the drug measured in the blood was 31,737.5 ng/mL in the younger group (6 to under 12 years) and 28,300.0 ng/mL in the older group (12 to under 18 years). In both age groups, this peak level was reached at 1 hour after the dose. The overall exposure to the drug over time (a measure of how much drug the body was exposed to in total) was reported as 69.4 h·mg/L in the younger group and 71.6 h·mg/L in the older group. The time it took for the drug level in the blood to fall by half was approximately 3.7 hours in the younger group and 3.6 hours in the older group. Regarding how much of the drug was removed through urine, the reported amounts were 227.9 mg in the younger group and 369.7 mg in the older group, with the rate at which the kidneys cleared the drug reported as 3.6 L/h and 5.4 L/h 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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• NCT00004412 · results posted 31 March 2015

  According to the results reported on ClinicalTrials.gov, this trial looked at a treatment called Arginine Butyrate (given through a drip into a vein) compared with standard local wound dressings, for people with leg or skin ulcers. A total of 23 participants started the first phase of the trial — 11 in the standard dressing group and 12 in the Arginine Butyrate group. The trial then had a crossover phase, where some participants from the standard dressing group could switch to the Arginine Butyrate treatment. The main thing the trial was measuring was whether an ulcer shrank by at least 25% of its original size, which was counted as "healed" for the purposes of this study. The reported data shows that, for the primary measure (ulcer shrinking by at least 25%), 24% of ulcers in the standard local care dressing group met this threshold, compared with 78% in the Arginine Butyrate group. For the secondary measure — the percentage of ulcers that closed completely within three months — the reported figures were 8% in the standard dressing group and 31% in the Arginine Butyrate group. These are the numbers as submitted to the registry; no further breakdown by individual patient characteristics was 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.

  Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗

• NCT01077921 · results posted 22 January 2015

  According to the results reported on ClinicalTrials.gov, this trial looked at a medicine called propranolol in people with sickle cell disease. It used a "crossover" design, meaning participants took propranolol for a period and a dummy pill (placebo) for another period, with a washout break in between. A total of 27 people were enrolled across two groups — 14 started on propranolol first and 13 started on placebo first. By the end of the trial, 22 participants completed both treatment periods. The main thing being measured was how "sticky" sickle cell red blood cells were — specifically, how much they stuck to the walls of blood vessels in a laboratory test. Several blood markers related to blood vessel activity were also measured as secondary outcomes. The reported data shows that for the primary outcome — the change in red blood cell stickiness at three different flow speeds (1, 2, and 3 units of pressure) — the numbers were close to zero or small in both the propranolol and placebo groups across all conditions tested. For example, at the lowest flow speed, the reported change was 0% for propranolol and −0.3% for placebo in unstimulated cells, and 7.4% versus 2.7% in stimulated cells. Similar small numerical differences were seen at the other two flow speeds. For the secondary outcomes — blood markers linked to blood vessel activity (called sE-selectin, sP-selectin, and sICAM-1) — the reported changes also varied between groups. For instance, sE-selectin changed by −3.9 ng/ml in the propranolol group and +3.7 ng/ml in the placebo group, while sP-selectin changed by −5 ng/ml and −12.8 ng/ml respectively, and sICAM-1 by −6.4 ng/ml and +5.4 ng/ml. No further interpretation of these numbers was reported in the structured 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.

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

• NCT01736657 · results posted 14 July 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 73 people with sickle cell disease, 72 of whom completed the study. The trial was testing a medical device called the Spectra Optia system, which is used to perform a red blood cell exchange — a procedure where a patient's red blood cells are removed and replaced with donor red blood cells. The main thing being measured was how closely the device's predictions about the exchange matched what actually happened in the patient's blood. The reported data shows that the key measurement — comparing the device's predicted outcome to the actual outcome in 60 participants — produced a ratio of 0.9. The trial had set a target range of 0.75 to 1.25, and this result falls within that range. For the secondary measurements, the reported data shows that in those same 60 participants, the device was recorded as completing the procedure and achieving a satisfactory exchange in 100% of cases, as judged by the treating doctors. The ratio of the patient's actual blood thickness (called haematocrit) after the procedure compared to what the device had targeted was reported as 1.03, meaning the two figures were very close to each other. The reported data also shows that among all 72 participants who completed the trial, zero participants experienced a serious harmful event that was considered related to the device. It should be noted that this figure covers device-related serious events only, and any other events during the trial are 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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• NCT01036802 · results posted 21 February 2014

  According to the results reported on ClinicalTrials.gov, this trial (NCT01036802) enrolled a very small number of participants — just 3 people in total, with 2 assigned to receive warfarin (a blood-thinning medication) and 1 assigned to receive a placebo (an inactive treatment). All 3 participants completed the trial. The trial was measuring whether warfarin had any effect on blood pressure in the lungs (measured using a type of heart ultrasound called Doppler echocardiography), how far participants could walk in 6 minutes, and several blood markers related to clotting and blood vessel activity. The reported data shows that for the primary measurement — estimated pressure in the lung's main artery — the warfarin group had an average reading of around 41–46 mm Hg (millimetres of mercury, a standard unit for pressure) at various points during the study, while the placebo group had readings of around 37–44 mm Hg. For the 6-minute walk test, the reported data shows the warfarin group walked an average of around 1,275–1,381 feet and the placebo group around 1,075–1,135 feet at various time points. No deaths were recorded in either group during the study. The reported data shows that no results were submitted for several of the secondary measurements, including the blood markers for clotting activity, platelet activation, and blood vessel activation — these figures were not reported. It is important to note that with only 3 participants, this was an extremely small study, and the numbers above reflect averages across just 1–2 people per 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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• NCT00004143 · results posted 11 November 2013

  According to the results reported on ClinicalTrials.gov, this trial enrolled just 2 participants, and both completed the study. The trial was looking at a type of stem cell transplant (where blood-forming cells from a donor are given to a patient) and was measuring whether the donor cells successfully "took hold" in the body — a process called engraftment — as well as whether serious complications occurred and whether participants were still alive two years later. The reported data shows that both participants (2 out of 2) showed signs that the donor cells had successfully established themselves in the body, both for infection-fighting white blood cells (neutrophil engraftment) and for platelets (the cells that help blood clot). The reported data also shows that both participants (2 out of 2) experienced severe graft-versus-host disease (a serious condition where donor immune cells attack the recipient's body, graded 3–4 out of 4 on a severity scale). However, 0 out of 2 participants had unexpected serious side effects beyond what the study anticipated, and 0 out of 2 participants died from complications directly related to the transplant. Both participants were reported to be alive at the two-year mark. It is worth noting that with only 2 participants, this was an extremely small trial, and the reported figures should be understood in that context. The reported data does not allow for broad conclusions about how this treatment might perform across a wider 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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• NCT00508027 · results posted 16 August 2013

  According to the results reported on ClinicalTrials.gov, this trial looked at the effects of the medication simvastatin across three escalating (gradually increasing) dose levels in a single group of participants. A total of 20 people started at Dose Level 1, with 12 completing that phase. Another 20 started at Dose Level 2, with 16 completing it. Only 2 participants were enrolled at Dose Level 3, and both completed it. The trial was measuring changes in several substances found in the blood — including markers linked to inflammation and blood vessel activity — before and after participants took simvastatin. Because only 2 people reached Dose Level 3, the researchers did not analyse the results for that group. The reported data shows the following average changes in blood marker levels after treatment. For Dose Level 1 compared to Dose Level 2: nitric oxide metabolites (a chemical signal in the blood) changed by +7 versus +19.7 micromolar; a protein linked to inflammation called hs-CRP changed by −7.7 versus −3.6 mg/L; another inflammation marker called IL-6 changed by −0.6 versus −0.3 pg/mL; a marker related to blood vessel growth called VEGF changed by −164 versus −30 pg/mL; a molecule involved in cell stickiness called VCAM-1 changed by −44 versus −86 ng/mL; and a clotting-related marker called tissue factor changed by −9 versus −36 pg/mL. Negative numbers indicate a decrease from the starting level, and positive numbers indicate an increase. The reported data shows results only for Dose Levels 1 and 2, as the Dose Level 3 group was too small to analyse. No data for Dose Level 3 outcomes was reported for any of these blood markers. These numbers reflect the averages across participants at each dose level, and no further breakdown of the data was included in the submission. 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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• NCT00350844 · results posted 8 May 2013

  According to the results reported on ClinicalTrials.gov, this trial enrolled 6 participants, all of whom received a medicine called hydroxyurea. Five participants completed the study, while one did not finish. The trial was looking at a specific measurement related to blood flow in the heart — called tricuspid regurgitant jet velocity (TRJV), which is a way of checking for signs of high blood pressure in the lungs using an ultrasound of the heart. This measurement was taken after 6 and 12 months of treatment. The study also tracked things like how well participants stuck to their treatment schedule, any signs of side effects in blood tests, markers in the blood related to red blood cell breakdown and blood vessel health, and quality of life using a standard questionnaire designed for children. The reported data shows that no numerical results were submitted to ClinicalTrials.gov for either the primary outcome (the heart ultrasound measurement) or any of the secondary outcomes (such as blood test results or quality of life scores). Because these figures were not included in the structured results data, it is not possible to describe what the measurements showed — the outcome numbers 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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• NCT00153985 · results posted 12 March 2013

  According to the results reported on ClinicalTrials.gov, this trial enrolled 2 participants, both of whom completed the study. The trial was looking at a stem cell transplant procedure for people with severe haemoglobinopathies — serious inherited blood conditions that affect how the body makes haemoglobin (the protein in red blood cells that carries oxygen). Both participants were in a single group receiving the transplant. The primary thing the trial was measuring was whether donor stem cells successfully "took hold" in the recipient's body — a process called engraftment. This was checked by looking at blood cell counts and testing what proportion of blood-forming cells came from the donor at set timepoints after the transplant. The reported data shows that 2 out of 2 participants met the criteria for this outcome. The trial also tracked two secondary outcomes: signs of organ stress related to the conditioning medicines used before the transplant (Busulfex, fludarabine, and alemtuzumab), and whether participants developed a complication called graft vs. host disease (where donor cells can react against the recipient's body). The reported data shows that 2 out of 2 participants were assessed for each of these secondary outcomes, however the data does not include a breakdown of how many experienced these complications — only that both participants were evaluated. It is worth noting that with only 2 participants, this was an extremely small trial, and the numbers on their own are very limited in what they can tell us. 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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• NCT01430091 · results posted 6 November 2012

  According to the results reported on ClinicalTrials.gov, this trial enrolled 18 adult participants, and all 18 completed the study. The trial was measuring how the body absorbed a drug called prasugrel when it was taken in different forms and ways. Specifically, it compared a standard swallowed tablet against four different ways of taking an orally dissolving tablet (ODT) — placed on top of the tongue, placed on top of the tongue with a juice chaser, chewed and swallowed, or dissolved under the tongue. The trial tracked the active ingredient that prasugrel turns into in the body (called the active metabolite), looking at how much of it got into the bloodstream and how quickly. The reported data shows three main measurements for each of the five ways of taking the drug. The first measurement was the total amount of the active ingredient absorbed over time (think of it as the overall "exposure" to the drug). For the standard tablet this was 43.3 units (ng\*h/mL), and for the four ODT methods it was 42.7, 42.3, 41.0, and 42.0 units respectively — broadly similar figures across all five groups. The second measurement was the peak level of the active ingredient in the blood. The standard tablet reached a peak of 47.3 ng/mL, the ODT on top of the tongue reached 47.6 ng/mL, while the other three ODT methods reached lower peaks of 32.3, 38.3, and 41.9 ng/mL. The third measurement was how long it took to reach that peak level. For most methods this was either 0.50 hours (about 30 minutes) or 0.75 hours (about 45 minutes), with the standard tablet, ODT on the tongue, and ODT under the tongue all reaching their peak at 0.50 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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• NCT01178099 · results posted 15 February 2012

  According to the results reported on ClinicalTrials.gov, this trial (NCT01178099) enrolled 26 participants in total — 13 healthy volunteers and 13 people living with sickle cell disease. All 13 healthy participants completed the study, while 12 of the 13 sickle cell participants completed it (one did not finish). The trial was measuring how the body processed the blood-thinning medicine prasugrel — specifically, how much of its active ingredient (the form the body actually uses) entered the bloodstream, and how it affected platelet activity (platelets are tiny blood cells involved in clotting). The reported data shows that the amount of prasugrel's active ingredient that entered the bloodstream — measured in two ways (total exposure over time and peak level reached) — was broadly similar between the two groups across the doses tested. For total exposure, the reported figures ranged from around 36 to 72 units (ng·h/mL) across both groups and doses. For peak blood levels, figures ranged from roughly 38 to 68 units (ng/mL) across both groups and doses. The reported data also shows that platelet activity was measured before and after 12 days of taking the medicine. In the healthy participant group, platelet aggregation (clumping together) was reported to have changed by around −50 percentage points on one measure and −70 on another. In the sickle cell group, the reported changes were −33 and −47 percentage points respectively. A separate measure of platelet inhibition (how much clumping was reduced) was reported as 61% in healthy participants and 42% in the sickle cell 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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• NCT00178464 · results posted 4 January 2012

  According to the results reported on ClinicalTrials.gov, this trial enrolled 11 participants, all of whom were assigned to receive aspirin. Nine participants completed the trial, while two did not finish. The trial was measuring the occurrence of adverse events (unwanted health events that happened during the study) and serious adverse events, and whether these were related to aspirin. It also intended to track a range of secondary measures including recruitment rates, brain scans, blood flow tests, cognitive (thinking and memory) tests, and rates of stroke, pain crises, and acute chest crises — however, no numerical results for those secondary outcomes were reported in the data submitted to ClinicalTrials.gov. The reported data shows that across the primary outcome measures, a total of 6 serious adverse events and 9 serious adverse events were recorded in two separate tallies under the serious adverse events outcome, with three further categories each recording 0 events. For the general adverse events outcome, the reported data shows counts of 8, 3, 5, 1, and 2 events across five separate categories. The data as submitted does not provide labels explaining what each individual category or sub-group of events refers to, so it is not possible to describe what each of those specific numbers represents beyond what is listed 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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• NCT00492531 · results posted 6 June 2011

  According to the results reported on ClinicalTrials.gov, this trial looked at whether sildenafil (compared to a dummy pill, or placebo) had any effect on people with a condition involving high blood pressure in the lungs (pulmonary hypertension). A total of 74 people took part — 37 in the sildenafil group and 37 in the placebo group. However, a large number did not finish the trial: 22 people in the sildenafil group and 23 in the placebo group did not complete it, leaving only 15 and 14 participants respectively who finished. The trial ran for 16 weeks and measured several things, including how far participants could walk in 6 minutes, breathlessness scores, a blood marker called BNP (a substance the heart releases under stress), and a measurement of lung blood pressure using an ultrasound of the heart. The reported data shows that the main thing being measured — how far participants could walk in 6 minutes — changed from the start of the trial to week 16. In the sildenafil group, the average walking distance went down by 16 metres, while in the placebo group it went down by 7 metres. For the lung blood pressure measurement (assessed by a specific ultrasound reading called tricuspid regurgitant jet velocity, measured in metres per second), the reported figures across different time points were similar in both groups, ranging around 2.9 to 3.2. Breathlessness scores (rated on a 0–10 scale, where 0 means no breathlessness and 10 means the worst possible) and BNP blood levels were also reported across multiple time points, with figures appearing broadly similar between the two groups, though detailed breakdowns of when each measurement was taken 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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• NCT00235391 · results posted 2 May 2011

  According to the results reported on ClinicalTrials.gov, this trial enrolled 1,683 participants in total, divided into six age groups: children aged 2 to under 6 (97 people), 6 to under 12 (200 people), 12 to under 16 (172 people), adults aged 16 to under 50 (1,164 people), 50 to under 65 (43 people), and 65 and older (7 people). The trial was measuring how deferasirox — a medicine used to reduce excess iron in the body — was used and tolerated across these different age groups, tracking serious unwanted health events and any events that caused someone to stop or pause taking the medication. In total, 1,393 participants completed the study. The reported data shows that the primary focus was on tracking the number of participants who experienced deaths, serious adverse events (that is, significant unwanted health occurrences), or events that led to stopping or pausing the study medicine. According to the results reported on ClinicalTrials.gov, deaths during the study were recorded as follows: 0 in the youngest three age groups and the 50–65 group, 1 in the 12–16 age group, and 4 in the 16–50 age group. Serious adverse events were reported in 10 participants aged 2–6, 22 aged 6–12, 27 aged 12–16, 129 aged 16–50, 4 aged 50–65, and 2 aged 65 and older. For the secondary outcome, the reported data shows that researchers tracked changes in participants' blood ferritin levels (a measure of iron stored in the body) from the start to the end of the study, grouping results as "improved," "no change," or "worsened." The data as submitted to ClinicalTrials.gov appears to include figures for two of these categories, though the labelling of which category each figure belongs to was not clearly separated in the submitted data. Across all age groups combined, the numbers reported were in the range of hundreds of participants in each category, but a complete breakdown by improvement, no change, or worsening for every age group was not fully distinguishable from the data provided. Where the worsening category data was not explicitly separated, those figures were not reported in a way that allows them to be stated 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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• NCT00094887 · results posted 23 April 2010

  According to the results reported on ClinicalTrials.gov, this trial enrolled 75 participants in each group — one group inhaled nitric oxide and the other inhaled a placebo (an inactive substance used for comparison). The trial was studying people experiencing a vaso-occlusive crisis (VOC), which is a painful episode associated with sickle cell disease. The main thing the trial was measuring was how long it took for that pain crisis to resolve, defined as pain dropping to a manageable level, no longer needing injected pain relief, being able to walk (if walking was possible before the crisis), and both the patient/family and doctor agreeing the person could go home and manage with oral pain medicines if needed. The reported data shows that, on average, participants in the inhaled nitric oxide group took approximately 61.8 hours for their pain crisis to resolve, compared with approximately 55.2 hours in the placebo group. For the secondary outcomes, the reported average length of hospital stay was about 4.1 days in the nitric oxide group and about 3.1 days in the placebo group. Five participants in the nitric oxide group and seven in the placebo group went home within the first 24 hours. The reported data also shows that the total opioid (strong pain medicine) doses received were similar between groups. Eight participants in the nitric oxide group and seven in the placebo group required a blood transfusion due to a lung complication called acute chest syndrome or pneumonia. For re-admissions to hospital within 30 days, the data appears to show two sets of figures — one set recording zero re-admissions in both groups, and another recording 9 re-admissions in the nitric oxide group and 17 in the placebo group; the reason for two sets of figures was not explained 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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• NCT00530270 · results posted 31 December 2009

  According to the results reported on ClinicalTrials.gov, this trial enrolled 12 people in total — 6 received a steroid medication called dexamethasone, and 6 received a placebo (a dummy treatment with no active ingredient). The trial was looking at a lung complication called Acute Chest Syndrome (ACS), which can occur in people with sickle cell disease. The main thing being measured was how long people had symptoms of ACS or how long they stayed in hospital, whichever came first. Eleven of the 12 participants completed the trial (one person in the dexamethasone group did not finish). The reported data shows that, for the primary measure, the dexamethasone group had a logged score of 2.4 and the placebo group had a logged score of 3.5 (these numbers were recorded using a mathematical transformation to handle the wide variation in hours, so they are not directly in plain hours). For the secondary measures, the reported data shows the dexamethasone group stayed in hospital for an average of around 41.5 hours, compared to around 62.3 hours for the placebo group. The dexamethasone group needed supplemental oxygen for an average of about 17.5 hours versus about 41.2 hours in the placebo group. The time spent with low blood oxygen levels was reported as approximately 13.8 hours for the dexamethasone group and 38.4 hours for the placebo group. For pain ratings (measured on a 0–10 scale, where 0 means no pain and 10 means severe pain), the reported data shows changes from the start of the trial to discharge, but the data as submitted contains multiple measurements per group and a clear single summary figure for each group was not separately labelled, so a straightforward comparison cannot be stated with confidence from the data provided. It is worth noting that this was a very small trial with only 6 people in each group, which means these figures should be interpreted with considerable caution. 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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• NCT00528801 · results posted 26 November 2009

  According to the results reported on ClinicalTrials.gov, this trial enrolled 160 people with sickle cell disease (the "cases" group) and 52 people without the condition (the "controls" group). The trial was measuring brain and thinking (neurocognitive) differences between these two groups. Specifically, it looked at performance on a standard intelligence test, the presence of small areas of damage in the brain seen on brain scans (MRI), and the volume of brain tissue in the outer layer of the brain (called cortical grey matter). Not everyone finished the study — 133 cases and 41 controls completed it. The reported data shows the following numbers for the two groups. On the thinking skills test (called the WAIS-III Performance IQ, where a score of 100 is considered average for the general population), the cases group scored an average of 90.6 points, while the controls group scored an average of 95.9 points. For the brain scan findings, 19 people in the cases group and 1 person in the controls group were reported to have small areas of damage in the brain known as lacunae (tiny spots, roughly 3–15 mm across, in certain deep brain regions). The reported data also shows that the average volume of the outer brain tissue was 543.5 mL in the cases group and 569.7 mL in the controls group. These numbers are simply what was measured and recorded during the study — they describe differences observed between the two groups at the time of testing. No data was reported on what these differences might mean over time or in other circumstances. 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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• NCT00513617 · results posted 4 August 2009

  According to the results reported on ClinicalTrials.gov, this trial (NCT00513617) enrolled 109 participants across three groups: 36 received a low dose of the treatment, 35 received a high dose, and 38 received a placebo (an inactive substance used for comparison). Not everyone finished the study — 25, 25, and 30 participants completed it in the low dose, high dose, and placebo groups respectively. The trial was measuring several things in the blood related to how red blood cells behave, including the activity of a channel in red blood cell walls (called the Gardos channel), levels of a gas called nitric oxide in the blood, and the concentration of a substance inside red blood cells called mean corpuscular haemoglobin concentration (essentially how packed with haemoglobin each red blood cell is). The reported data shows the following changes from the start of the study to the end. For Gardos channel activity (measured in units of mmol per a very large number of cells per minute), the low dose group showed a change of −0.034, the high dose group showed +0.004, and the placebo group showed +0.108. For nitric oxide levels in the blood (measured in micromoles), the low dose group showed a change of −3.87, the high dose group showed +2.93, and the placebo group showed −3.03. For the haemoglobin concentration inside red blood cells (measured in grams per decilitre of blood), the low dose group showed a change of −1.85, the high dose group showed +0.77, and the placebo group showed −0.07. Three other secondary outcomes — a molecule called sVCAM, a marker of cellular damage called 8-iso-PGF2a, and a substance called endothelin-1 — were listed as planned measures but no results data was reported for these 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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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.