Reported trial results for Spinal Muscular Atrophy

50 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

• NCT05430113 · results posted 16 March 2026

  According to the results reported on ClinicalTrials.gov, this trial looked at the use of spinal cord stimulation (a technique that delivers mild electrical signals to the spine) in a very small group of 3 participants. All 3 people completed the study. The trial was measuring whether turning the stimulation on made any difference to leg muscle strength, movement, walking distance, and fatigue, compared to when the stimulation was turned off. The reported data shows that for the main (primary) measure of muscle strength — specifically the force produced at the hip — 2 out of 3 participants met the pre-set target of at least a 20% increase in force when the stimulation was on compared to off. A separate result for the same measure appears to show 3 out of 3, though the data as submitted lists two separate figures (2 and 3) without further explanation of the difference. For the other primary measure, adverse events (unwanted or harmful reactions), 0 out of 3 participants reported a serious or intolerable event related to the stimulation. For the secondary measures, the reported data shows: 1 out of 3 participants met the target for improved range of movement at the hip and knee; 2 out of 3 met the target for changes in muscle activation signals; 2 out of 3 met the target for walking a greater distance during a 6-minute walk test; and 3 out of 3 participants showed a change in fatigue during the movement tests. It is worth noting this was a very small study of only 3 people, so the numbers above represent individual people rather than large 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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• NCT06411912 · results posted 3 March 2026

  According to the results reported on ClinicalTrials.gov, this trial (NCT06411912) enrolled 54 people in total — 26 received a placebo and 28 received the investigational drug NIDO-361. The trial was studying a condition called Spinal and Bulbar Muscular Atrophy (SBMA), a rare inherited muscle-wasting disease. The main things being measured were changes in lean muscle volume across the thighs and whole body (assessed by how much fat had replaced muscle tissue), as well as how many participants experienced unwanted health events (adverse events) during the study. Secondary measurements included scores on a functional rating scale and distances walked in two- and six-minute walking tests. The reported data shows that for lean muscle volume — specifically the healthiest category of muscle (low fat content, called "A-muscles") — the thigh muscles in the placebo group measured around 4.14 cm³ on average, compared to around 3.86 cm³ in the NIDO-361 group at the start. Small changes over the study period were recorded in both groups across all muscle categories, but the specific figures reported were close between the two groups. For the walking tests, the reported data shows the placebo group started with an average 2-minute walk distance of about 128 metres and the NIDO-361 group about 113 metres; over 360 days, the placebo group's distance changed by approximately −1.8 metres while the NIDO-361 group's changed by approximately +3.3 metres. For the 6-minute walk test, the placebo group changed by about −4.6 metres and the NIDO-361 group by about +8.9 metres over 360 days. On the functional rating scale (scored 0–30, where higher is better), both groups started around 15–16 points; after 360 days, the placebo group's score changed by about +1.2 points and the NIDO-361 group's by about +0.1 points. Regarding unwanted health events, 25 of 26 placebo participants and 26 of 28 NIDO-361 participants reported at least one adverse event; serious adverse events were reported by 9 placebo participants and 17 NIDO-361 participants; 3 participants in each group discontinued the study drug; and one death was reported in the NIDO-361 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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• NCT05089656 · results posted 8 December 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT05089656) enrolled 126 children aged 2 to under 18 years with spinal muscular atrophy (SMA). It was a crossover trial, meaning participants were split into two groups: 75 children received OAV101 (an investigational gene therapy) first for 52 weeks, followed by a sham (inactive) procedure, while 51 children received the sham procedure first, then OAV101. The main thing being measured was change in a motor ability score called the HFMSE — a 66-point scale used specifically in SMA where higher scores mean greater physical ability. The reported data shows that after the first 52-week period, children who received OAV101 first had an average increase of 2.39 points on the HFMSE scale, while those who received the sham procedure first had an average increase of 0.51 points. Among younger children aged 2 to under 5, the reported average increases were 3.00 points (OAV101 group) and 1.56 points (sham group). For upper limb motor ability (measured on a separate 37-point scale called the RULM), the reported average increases across all ages were 2.44 points for the OAV101 group and 0.92 points for the sham group. Looking at the proportion of children who improved by at least 3 points on the HFMSE, the reported data shows 39.2% in the OAV101 group compared with 26.0% in the sham group across all ages, and 48.8% versus 37.9% in the younger age 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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• NCT04090528 · results posted 5 December 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04090528) enrolled 60 people with prostate cancer, split evenly into two groups of 30. One group received a single DNA vaccine (called pTVG-HP) combined with a drug called pembrolizumab, while the other group received two DNA vaccines (pTVG-HP and pTVG-AR) combined with pembrolizumab. The main thing the trial was measuring was how many people in each group had not seen their cancer get worse, and had not died, at the six-month mark — a measure known as "progression-free survival rate." By the end of the study, 21 people in the one-vaccine group and 17 people in the two-vaccine group had completed the trial, with the remainder not completing it for various reasons. The reported data shows that in the one-vaccine group, 51% of participants were free from disease progression at six months. In the two-vaccine group, 45% of participants were free from disease progression at six months. Before the trial started, researchers had hoped the two-vaccine group would reach at least 55%, compared to an expected 20–30% for the one-vaccine group, so the reported figures can be seen in that context — though what this means clinically is a matter for medical professionals to interpret. The reported data shows that results for the secondary outcome measures — including overall response rate, PSA (a prostate-related protein) response rate, median progression-free survival, duration of response, and overall survival — were not reported in the data submitted to ClinicalTrials.gov, so those figures cannot be described 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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• NCT02176863 · results posted 5 December 2025

  According to the results reported on ClinicalTrials.gov, this trial tested a medicine called Flebogamma® 5% DIF (an intravenous immunoglobulin, a product made from donated blood plasma) against a placebo (an inactive treatment) in people with a particular condition affecting physical function. The trial was run in two stages and used different doses — 2 grams per kilogram of body weight and 1 gram per kilogram. In total, 191 people started the trial across all five groups: 42 in each of the three Stage 1 groups and 33 and 32 in the two Stage 2 groups. The number who completed the trial varied by group, ranging from 25 to 34 in Stage 1 and 26 in each Stage 2 group. The main thing being measured was how far participants could walk in two minutes (called the Two-Minute Walk Distance test), with a greater distance meaning better physical performance. The reported data shows that at the start of the trial, participants in the five groups could walk between roughly 101 and 113 metres in two minutes. Over the course of the trial, the reported change from that starting point (meaning how much further or less far people walked compared to when they began) was: +7.50 metres for the Stage 1 high-dose group, +10.89 metres for the Stage 1 lower-dose group, +3.17 metres for the Stage 1 placebo group, +11.31 metres for the Stage 2 lower-dose group, and +5.65 metres for the Stage 2 placebo group. For the secondary outcomes — which were additional things being measured — the reported data shows that on a pain scale from 0 (no pain) to 100 (worst pain), the change from the starting point was −3.2 for the Flebogamma group and −2.6 for the placebo group (a negative number meaning slightly less pain reported). On a physical health quality-of-life score (where higher is better), the change from baseline was +4.78 for the Flebogamma group and +2.79 for placebo. For a six-minute walk test measuring endurance, the Flebogamma group reported walking 29.16 metres further than at the start, compared with 13.36 metres further 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.

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• NCT02386553 · results posted 20 October 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT02386553) enrolled 25 infants who had been diagnosed with spinal muscular atrophy (SMA) before they showed any symptoms — meaning they were identified through genetic testing rather than because they were already unwell. The babies were divided into two groups based on how many copies they had of a gene called SMN2 (15 babies with 2 copies, and 10 with 3 copies), as this gene affects how the condition may develop. The trial was testing a medicine called ISIS 396443 (also known as nusinersen) and was primarily measuring the time until a child either died or needed significant breathing assistance. Secondary measures included whether children developed signs of SMA, whether they were alive at the end of the study, and whether they reached normal movement milestones such as sitting, standing, and walking. The reported data shows that the primary outcome — time to death or need for breathing support — was listed as "NA" (not available/not reported) in the submitted results, so a specific figure for this measure cannot be described. For the secondary outcomes, the reported data shows that 100% of participants in both groups were alive at the time of reporting. Regarding whether children developed clinically recognised signs of SMA, the reported proportion was 0.67 (meaning about 67 in every 100) in the 2-copy group and 0.20 (20 in every 100) in the 3-copy group. For motor development milestones assessed using standard WHO movement checks (such as sitting without support or walking alone), the reported data shows that between 87% and 100% of children in the 2-copy group, and 100% of children in the 3-copy group, reached each of the six milestones. A muscle strength and function score (rated 0–64, where higher means better) showed a reported change from a starting score of around 47 in the 2-copy group and around 52 in the 3-copy group, with one additional figure of 29 reported, though the data as submitted did not make clear which time point this last figure relates to. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03032172 · results posted 1 October 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT03032172) enrolled a total of 174 participants across four treatment groups during the main treatment period: 13 received RO6885247, 76 received nusinersen, 71 received olesoxime, and 14 received AVXS-101. The trial was an observational study tracking participants who were already receiving one of these four medicines for spinal muscular atrophy (SMA). After the main treatment period, those who completed it could continue into an open-label extension phase — meaning everyone knew which medicine was being taken — with 154 participants entering that phase across all four groups. The trial was primarily measuring safety-related experiences, including any unwanted medical events, changes in puberty development, neurological findings, changes in body weight, and any signs of suicidal thoughts. The reported data shows that across the treatment period, nearly all participants in each group experienced at least one adverse event (an unwanted medical occurrence of any kind, whether or not considered related to the medicine): 12 out of 13 in the RO6885247 group, 76 out of 76 in the nusinersen group, 70 out of 70 in the olesoxime group, and 14 out of 14 in the AVXS-101 group. Only 1 participant — in the nusinersen group — stopped treatment because of an adverse event. Regarding puberty development, 3 participants in the olesoxime group were recorded as having a shift in their puberty status from what was expected for their age. For neurological findings beyond those expected with SMA, 1 participant each in the nusinersen and AVXS-101 groups were reported to have such a condition. The reported data also shows that small numbers of participants across groups had scores indicating some level of suicidal thoughts on the rating scale used: 1 in the RO6885247 group, 5 in the nusinersen group, and 2 in the olesoxime group, with none in the AVXS-101 group. Changes in body weight from the start of the study varied across groups and across different time points; some groups showed small increases and others small decreases, though not all weight data was available for every group at every time point, and where figures were not reported the data was not 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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• NCT04089566 · results posted 5 June 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT04089566) looked at nusinersen, a medicine used for spinal muscular atrophy (SMA) — a condition that affects muscle strength and movement. The trial was run in three parts (A, B, and C) and tested different dose levels of the medicine. In total, 145 people took part across all groups. Part A included 6 people, Part B included 99 people (split between those with an early-onset and later-onset form of SMA), and Part C included 40 people. Not everyone who started the trial completed it — for example, in one of the Part B infantile-onset groups, 12 out of 25 participants did not complete the study. The reported data shows that one of the main things measured in Part B (for infants with early-onset SMA) was a motor skills test called the CHOP-INTEND, which scores muscle strength and movement on a scale from 0 (worst) to 64 (best). According to the results reported on ClinicalTrials.gov, infants who received the higher dose of nusinersen (50/28 mg) had an average change in score of 42.9 points from the start of the study to around six months later, compared to an average change of 16.9 points in a comparison group from a separate study who received a sham (inactive) procedure. For Parts A and C, the trial also tracked unwanted medical events. The reported data shows that in Part A (6 participants), 4 experienced an unwanted medical event and 1 experienced a serious one; in Part C (40 participants), 37 experienced an unwanted medical event and 6 experienced a serious one. The trial also tracked changes in blood, urine, and spinal fluid measurements, with varying numbers of participants showing shifts in these values 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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• NCT05386680 · results posted 3 June 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT05386680) enrolled 27 participants who received a single dose of the gene therapy OAV101 (at a dose level of 1.2×10¹⁴ viral genome particles). Of the 27 who started, 25 completed the study and 2 did not. The trial was primarily focused on tracking unintended medical events (called adverse events) that occurred after treatment, and secondarily on measuring changes in participants' physical abilities over 52 weeks using two standard movement assessment tools used in spinal muscular atrophy (SMA). The reported data shows that, out of 27 participants, 27 experienced at least one treatment-emergent adverse event (an unintended medical occurrence that happened after receiving the treatment). Thirteen participants experienced adverse events considered related to the treatment. The trial also tracked specific categories of concern — including liver-related events, low platelet counts, heart-related events, and nerve-related signs — with small numbers of participants (ranging from 1 to 8 across different categories) recorded under these special-interest groups. The standard deviations and full breakdown labels for many of these sub-counts were not fully reported in the submitted data. The reported data shows that, on the movement assessment scales measured at 52 weeks, participants' scores on the Hammersmith Functional Motor Scale Expanded (HFMSE — a 0–66 motor ability score) changed by an average of 0.17 points from where they started (with a separate statistical estimate of 1.05 points). On the Revised Upper Limb Module (RULM — a 0–37 upper limb ability score), the average change from baseline was 0.29 points. No standard deviation or margin-of-error figures were included in the submitted results for these measures. These numbers describe what was recorded in this group; they do not tell us what the results mean for any individual. 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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• NCT04926181 · results posted 15 May 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled just 2 participants, both of whom completed the study. The trial was testing a combination of two medicines — apalutamide and cetrelimab — in a single group, with no comparison group. The main thing the trial set out to measure was a "composite response rate" — meaning whether participants showed both a significant drop in a prostate cancer marker in the blood (called PSA) and a shrinkage of tumours visible on scans. The trial also tracked a number of secondary measures, including side effects linked to the treatment, how long participants went without their cancer visibly progressing on scans or by PSA levels, and how many participants had large drops in their PSA readings. The reported data shows that the primary outcome — the composite response rate — had no numbers submitted to ClinicalTrials.gov, so those results were not reported. For the secondary outcomes, the reported data shows that all participants (a proportion of 1.00, meaning both of the 2 participants) experienced at least one side effect considered to be related to the study treatment. Half of the participants (a proportion of 0.50, meaning 1 out of 2) were reported to have had a drop in PSA of 50% or more from their starting level, and likewise half (again 1 out of 2) were reported to have had a drop of 90% or more. The two remaining secondary outcomes — median time without disease progression on scans, and median time without PSA progression — had no figures submitted and were not reported. It is worth noting that with only 2 participants, this trial was extremely small, and the reported numbers reflect the experience of just 2 individuals. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT02268552 · results posted 9 April 2025

  According to the results reported on ClinicalTrials.gov, this trial tested a medicine called LMI070 in people with a condition the trial was designed to study. The trial ran in two main parts plus an extension phase. In Part 1, small groups of 2–4 participants each received one of five different doses of LMI070 (measured in milligrams per square metre of body surface area), giving a total of 13 participants in that part. In Part 2, 25 participants were divided into two groups receiving different doses measured by body weight (0.625 mg/kg and 2.5 mg/kg). The trial was measuring, among other things, whether certain medically significant unwanted reactions — called dose-limiting toxicities — occurred in the first 14 days, and also tracking any unwanted events (called adverse events) that appeared or worsened after participants started the medicine. The reported data shows that in Part 1, zero participants in any of the five dose groups experienced a dose-limiting toxicity during the first 14 days. For unwanted events that emerged during treatment (across all groups in both parts), the reported data shows that all participants in every group experienced at least one such event — that is, 2 out of 2, 2 out of 2, 2 out of 2, 4 out of 4, and 3 out of 3 participants in the five Part 1 dose groups, and 10 out of 10 and 15 out of 15 participants in the two Part 2 dose groups. The trial also measured how the medicine moved through the body (called pharmacokinetics). The reported data shows that as the dose increased across the five Part 1 dose levels, the peak concentration of the medicine detected in the blood ranged from roughly 9 ng/mL at the lowest dose up to about 97 ng/mL at the highest dose, and the total amount of medicine the body was exposed to over time also increased with each higher dose level. 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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• NCT02594124 · results posted 22 October 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT02594124) enrolled a total of 292 participants across nine groups. The participants had either infantile-onset or later-onset Spinal Muscular Atrophy (SMA) and came from several earlier related studies. The groups included people who had previously received a placebo-like "sham" procedure, people who had previously received the study drug nusinersen (also referred to as ISIS 396443), and people entering from other studies. The trial was measuring a range of monitored observations — including unwanted medical events (called adverse events), abnormalities in vital signs (such as blood pressure and heart rate), body weight changes, neurological (nerve and muscle) examination findings, and blood test results. The reported data shows that across the nine groups, the number of participants who experienced any treatment-emergent adverse event (that is, a new or worsening medical event that appeared after dosing began) ranged from 8 out of 8 participants in one group up to 80 out of 83 participants in another group. Serious adverse events — defined as events involving death, being life-threatening, requiring hospitalisation, or causing significant long-term disability — were also counted, though the full breakdown across all nine groups was not completely reported in the data provided. For the other monitored areas: abnormalities in vital signs recorded as adverse events were reported in very small numbers across most groups (ranging from 0 to 6 participants per group); weight changes of 7% or more were reported in 0 to 3 participants per group; neurological examination abnormalities recorded as adverse events were reported in 0 to 2 participants per group; laboratory test abnormalities were reported in 0 to 6 participants per group; and abnormalities in blood clotting measures were reported in 0 to 2 participants 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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• NCT01645787 · results posted 3 September 2024

  According to the results reported on ClinicalTrials.gov, this trial enrolled 11 people in total — six in one group and five in the other. It used a "crossover" design, meaning everyone took turns receiving the study drug (4-aminopyridine, also known as Ampyra) and a placebo (a dummy pill with no active ingredient), so each person experienced both treatments at different points. The trial was looking at whether the drug made any difference to walking ability and muscle function in people with Spinal Muscular Atrophy (SMA) Type 3, a condition that affects muscle strength and movement. The study ran across several periods — short-term phases of about two weeks and longer-term phases of about six weeks — with rest periods in between. The reported data shows that the main thing measured was how far participants could walk in six minutes (called the Six Minute Walk Test). In the short-term phase, those taking the study drug walked an average of about 293 metres, compared to about 300 metres for those on placebo. In the longer-term phase, those on the study drug walked an average of about 299 metres, compared to about 295 metres for those on placebo. For the secondary measures, a motor function scoring scale (rated 0–66, with higher being better) showed scores of around 50.8 on the drug versus 50.4 on placebo (short term), and about 50.9 versus 50.3 (long term). A muscle strength test (scored 0–280, higher being better) showed scores of roughly 181.9 on the drug versus 182.2 on placebo (short term), and about 177.8 versus 181.1 (long term). The reported data does not include any statistical analysis of these differences, so it is not possible to draw conclusions from the numbers alone. It is also worth noting that with only 11 participants, this was a very small trial, and one participant did not complete one of the study periods. 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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• NCT05073133 · results posted 31 May 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT05073133) involved 16 participants who received a treatment called OAV101. The trial was primarily measuring the number of participants who experienced medical events (called adverse events, or AEs) after receiving the treatment — that is, any unwanted sign, symptom, or health change that occurred during the study period. It also tracked a set of specific health concerns the researchers were watching closely, and as a secondary measure, it looked at how many children reached certain movement milestones (such as sitting, crawling, standing, and walking) based on a World Health Organization checklist. The reported data shows that of the 16 participants who started the trial, 14 completed it and 2 did not. For the primary outcomes, the reported numbers show that 16 participants had at least one adverse event recorded, with 11 participants recorded under several of the specific adverse event categories, and 12 participants recorded in another category — though the data as submitted does not clearly label each individual number to a specific category name, so a full breakdown cannot be provided here. For the specially monitored health concerns (including liver-related events, low platelet counts, heart-related events, nerve-related sensory changes, and a condition affecting small blood vessels), the reported figures ranged from 1 to 11 participants across the various categories. For the movement milestones, the reported data shows that 6 participants reached the milestone of sitting without support, 2 reached hands-and-knees crawling, 1 reached standing with assistance, and none were recorded as reaching walking with assistance, standing alone, or walking alone at the relevant time points assessed. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03779334 · results posted 5 March 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT03779334) looked at a medicine called risdiplam in infants who had been diagnosed with spinal muscular atrophy (SMA) before they showed any symptoms — meaning they were identified through newborn screening. A total of 26 babies took part, split into three groups based on how many copies of a gene called SMN2 they carried: 8 babies had 2 copies, 13 had 3 copies, and 5 had 4 or more copies. The trial's main question was focused specifically on a smaller subgroup of 5 babies from the 2-copy group who met particular criteria at the start of the study. Notably, the reported data shows that none of the 26 participants completed the study as planned — all are listed as "not completed," though the reasons for this are not detailed in the data provided here. The reported data shows that for the primary outcome — whether babies in that specific 5-person subgroup could sit without any support for at least 5 seconds — 80% of those participants (that is, 4 out of 5 babies) achieved this milestone. This was the only outcome measure for which numbers were reported. For all of the secondary outcomes — including things like whether participants developed SMA symptoms, how many remained alive without needing a breathing machine, and whether babies reached other movement milestones — no numerical results were provided in the data submitted to ClinicalTrials.gov. Because most of the secondary outcomes have no reported figures, it is not possible to describe what those measurements found. The data was simply not reported for those measures. 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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• NCT04851873 · results posted 5 January 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT04851873) enrolled 24 children in total, divided into three groups based on body weight: 7 children weighing 8.5–13 kg, 8 children weighing more than 13–17 kg, and 9 children weighing more than 17–21 kg. All 24 children completed the study. Each child received a single dose of OAV101 (a gene therapy), and the trial was primarily measuring how many participants experienced unwanted medical events (called adverse events) and changes in vital signs such as blood pressure and breathing rate across the different weight groups. The reported data shows that all 24 participants across the three weight groups experienced at least one adverse event (sometimes called an "AE" — an unwanted medical occurrence during the study). When it came to serious adverse events (more significant unwanted medical occurrences), 8 out of 24 participants were reported to have experienced one — 1 in the lightest group, 4 in the middle group, and 3 in the heaviest group. The trial also tracked a specific list of pre-identified medical concerns, including effects on the liver, low platelet counts, heart-related events, nerve-related effects, and a condition affecting small blood vessels. The reported data shows 20 out of 24 participants experienced at least one of these pre-identified concerns, and 3 out of 24 experienced what were categorised as serious versions of these specific concerns. For blood pressure and breathing rate, the reported changes from the start of the study to later measurements were generally small and varied across the groups; the exact figures differed by weight group and time point, and no single consistent direction of change was seen across all 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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• NCT03819660 · results posted 30 November 2023

  According to the results reported on ClinicalTrials.gov, this trial enrolled 13 participants, all of whom completed the study with no drop-outs. The trial was looking at a medicine called amifampridine phosphate in people with Spinal Muscular Atrophy (SMA) Type 3 — a condition that affects muscle strength and movement. The study was primarily tracking how many participants experienced unwanted side effects (called treatment-emergent adverse events) over the long term, and it also measured changes in participants' quality of life using a standard questionnaire. The reported data shows that 5 out of the 13 participants experienced at least one treatment-emergent adverse event — that is, an unwanted health event that occurred during the time they were taking the medicine. For quality of life, the trial used a scoring tool called the INQoL questionnaire, where a higher score means a worse outcome, on a scale from 0 to 100. The reported data shows two scores for the group: 33.737 and 39.012. The trial record does not explain which time points these two scores correspond to, so the context for comparing them is not fully reported in the data. It is worth noting that with only 13 participants, this was a very small study. The trial did not include any paediatric (child) patients, so quality-of-life data was only collected using the adult version of the questionnaire. No further breakdown of the outcome numbers was reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03653390 · results posted 21 November 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03653390) involved 505 people in total across three groups: 183 people in the "EnhanceWellness for Disability" (EW-D) programme, 159 in a "Wellness Education" group, and 163 in a control group (who received neither programme). The trial was measuring whether taking part in these programmes made a difference to things like people's ability to join in community and social activities, their confidence in managing a long-term health condition, how much pain and fatigue got in the way of daily life, their psychological resilience, and how often they left home. Most participants completed the study, with 163, 150, and 155 finishing in each group respectively. The reported data shows the following changes in scores from the start of the trial. For the main measure — ability to participate in social and community activities (scored from 8 to 40, where higher means better) — the EW-D group's average score went up by 2.49 points, the Wellness Education group's went up by 1.25 points, and the control group's went up by 0.09 points over six months. For confidence in managing a chronic illness (scored 6–30, higher is better), the reported changes at three months were +2.19 for EW-D, +0.01 for Wellness Education, and −0.07 for the control group. For how much pain interfered with daily life (scored 4–20, where higher means more interference), the changes at three months were −1.22, −0.55, and −0.23 respectively — meaning all groups reported some reduction. For fatigue interference (same scale), the changes were −1.89, −1.09, and −0.76. For psychological resilience (scored 0–40, higher is better), the changes were +0.44, +0.28, and −0.77. Finally, for the average number of trips outside the home per day measured over 12 months, the reported changes were −0.25 for EW-D, −0.12 for Wellness Education, and +0.03 for the control 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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• NCT03381729 · results posted 16 February 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03381729) enrolled 32 children with spinal muscular atrophy (SMA) across several groups, divided by age and by the dose of the gene therapy AVXS-101 (also known as onasemnogene abeparvovec) they received. The younger children were aged 6 months to under 2 years, and the older children were aged 2 years to under 5 years. All 32 children who started the trial completed it. The trial was measuring things like the ability to stand or walk independently, changes in a motor function score, how many children experienced medical events after receiving the treatment, and how many hours per day some children needed breathing support. The reported data shows the following for the younger age group (6 months to under 2 years): in the lowest-dose group, 1 out of 3 children achieved the ability to stand alone unsupported for at least 3 seconds, and 2 out of 3 achieved the ability to take at least 5 independent steps; in the middle-dose group, 1 out of 13 achieved standing and 12 out of 13 achieved walking; in the highest-dose group, 0 out of 4 achieved standing and 4 out of 4 achieved walking. For the older age group (2 years to under 5 years) in the middle-dose group, a motor function score (rated from 0 to 66, where higher means better ability) changed by an average of 6.0 points above where it started after 12 months. Regarding medical events that occurred after receiving the treatment, all participants in each group experienced at least one such event: 3 of 3 in the lowest-dose younger group, 13 of 13 in the middle-dose younger group, 12 of 12 in the middle-dose older group, and 4 of 4 in the highest-dose younger group. Serious medical events were reported in 1, 2, 4, and 0 participants in those same groups respectively. For breathing support, data was only reported for some groups — the older middle-dose group started at an average of around 10.5 hours per day and the figures varied across different time points, while the data was not reported for the youngest and highest-dose groups in the same way. 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 ↗

• NCT03921528 · results posted 17 November 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT03921528) looked at a treatment for Spinal Muscular Atrophy (SMA) across three groups of participants, divided by their type of SMA and mobility level. In total, 58 people started the trial: 11 in Cohort 1 receiving the study drug alone, 12 in Cohort 1 receiving it alongside another SMA treatment, 15 in Cohort 2, and 10 each in two Cohort 3 groups (a lower dose and a higher dose). The trial measured changes in physical ability scores over 12 months using standardised movement assessments — tools that assign a number to how well a person can perform various physical tasks, where a higher score means better physical ability. The reported data shows the following changes in those physical ability scores after 12 months. For Cohort 1 (participants who could walk, aged 5–21), the average score on a movement scale (out of 69) changed by −0.1 points for those on the study drug alone, and 0.0 points for those also on another SMA treatment — meaning scores stayed roughly the same as at the start. For Cohort 2 (non-walking participants aged 5–21), the average score on a different movement scale changed by +0.6 points. For Cohort 3 (non-walking participants aged 2 and older), the average score changed by +5.3 points in the lower-dose group and +7.1 points in the higher-dose group. Regarding secondary measures in Cohort 1, the reported data shows that in a six-minute walking test, the monotherapy group walked an average of 20.6 metres less than at the start, while the dual therapy group walked an average of 11.0 metres more. In a 30-second sit-to-stand test, both groups showed small declines on 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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• NCT03986671 · results posted 21 June 2022

  According to the results reported on ClinicalTrials.gov, this trial enrolled a total of 50 participants across three groups: 36 people described as normal (healthy) subjects, 9 people with Amyotrophic Lateral Sclerosis (ALS, a neurological condition affecting muscle control), and 5 people with Obstructive Sleep Apnea (a condition where breathing repeatedly stops during sleep). The trial was measuring whether a sensor-based testing method called TM-EMG — a way of recording muscle activity in the throat and tongue — produced consistent results compared to an existing standard method (NEMG, another muscle-activity recording technique). Only a smaller number of participants passed a screening stage and went on to complete the full study: 6 healthy subjects, 5 with ALS, and 5 with sleep apnea. The reported data shows that the main thing being measured was how often two independent medical experts, who did not know each other's assessments, agreed when reviewing the TM-EMG results — a way of checking whether the test gives reliable, consistent readings. The reported agreement figures were 95.7% for the healthy subjects group, 78.1% for the ALS group, and 90% for the sleep apnea group. These percentages represent how often the two experts reached the same conclusion when looking at the same test results, with 100% meaning they always agreed. It is worth noting that the numbers who completed the study were quite small — particularly in the ALS and sleep apnea groups — which the reported data does not comment on further. No other outcome measures beyond this agreement percentage were reported in the structured results submitted to ClinicalTrials.gov. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03837184 · results posted 11 January 2022

  According to the results reported on ClinicalTrials.gov, this trial involved just 2 participants, both of whom completed the study. The trial was testing a treatment called AVXS-101 and was measuring two things: whether participants could sit up on their own for at least 10 seconds without support, and whether participants reached 14 months of age without dying, needing permanent breathing support, or withdrawing from the study. The reported data shows that 1 out of the 2 participants was able to sit upright without support for at least 10 seconds — this was confirmed by video recording using a standard international definition of independent sitting. For the second measure, the reported data shows that both participants (2 out of 2) reached 14 months of age without dying, without needing permanent ventilation (a machine to breathe for them continuously), and without leaving the study early. It is worth noting that this was a very small study with only 2 participants, so the numbers reported here represent a very limited group. No additional outcome data beyond these two measures was reported in the structured results submitted to ClinicalTrials.gov. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03505099 · results posted 11 January 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT03505099) involved 29 babies in total, split into two groups based on their genetic profile. Fourteen babies were in Cohort 1 (carrying two copies of a gene called SMN2) and fifteen were in Cohort 2 (carrying three copies of SMN2). All 29 participants completed the study. The trial was measuring whether babies reached certain physical milestones — such as sitting, standing, and walking — that are not typically expected in children with their condition. The reported data shows that for Cohort 1, all 14 participants were reported to have achieved sitting alone for at least 30 seconds without support (the primary goal for that group). For Cohort 2, all 15 participants were reported to have achieved standing alone for at least 3 seconds without support (the primary goal for that group). Turning to the secondary goals, the reported data shows that all 14 babies in Cohort 1 were alive, not on permanent breathing support, and still in the study at 14 months of age. Thirteen out of 14 babies in Cohort 1 were reported to have maintained a healthy weight without needing a feeding tube up to 18 months of age. For Cohort 2, 14 out of 15 participants were reported to have achieved walking alone, defined as taking at least 5 independent steps. 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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• NCT03461289 · results posted 2 April 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03461289) enrolled 33 children who received a treatment called onasemnogene abeparvovec-xioi. The trial was looking at two main things: first, how many children could sit up on their own for at least 10 seconds without using their arms or hands to balance (the primary, or main, goal); and second, how many children were still alive, not needing a permanent breathing machine, and still in the study by the time they reached 14 months of age. Thirty-two of the 33 children completed the study, and one did not. The reported data shows that 14 out of 33 participants achieved independent sitting for at least 10 seconds — the main outcome being tracked. For the secondary outcome (an additional measure the trial was monitoring), 31 out of 33 participants were reported as having reached 14 months of age without dying, without needing permanent ventilation (a breathing machine used full-time), and without withdrawing from the study. The reported data shows only these two numerical results — no further breakdown or additional outcome 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.

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• NCT01422200 · results posted 19 October 2020

  According to the results reported on ClinicalTrials.gov, this trial enrolled 22 participants in total — 11 in a group that received a flu vaccine by subcutaneous injection (under the skin) and 11 in a group that received the same vaccine by intramuscular injection (into the muscle). All 22 participants completed the study. The trial was measuring how the body's immune response — specifically the level of flu-fighting antibodies in the blood — compared between the two injection methods, across three different flu strains included in the vaccine. The reported data shows antibody responses using something called a "geometric mean titer ratio" — essentially a way of expressing how much antibody levels rose after vaccination compared to before. For the subcutaneous group, the reported ratios across the three flu strains were 3.76, 2.00, and 1.76. For the intramuscular group, the corresponding figures were 3.53, 1.46, and 2.00. A higher number indicates a greater rise in antibody levels between the pre-vaccination and post-vaccination measurements, though what these numbers mean clinically is not described in the submitted data. The reported data also includes counts of participants who experienced local reactions at the injection site (such as pain, redness, or swelling) or general body reactions (such as body aches, headache, or cough) in the four days after vaccination. These figures ranged from 0 to 5 participants per symptom category across the two groups, though a full breakdown of which numbers correspond to which specific symptoms was not clearly distinguishable in the data as 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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• NCT03988907 · results posted 19 October 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03988907) involved a total of 35 participants split into two parts — 8 people in Part 1 and 27 people in Part 2. All 8 participants in Part 1 completed the study, as did 26 of the 27 in Part 2 (one person did not complete Part 2). The trial was measuring how a drug called risdiplam — used for spinal muscular atrophy — affects the way the body processes another drug called midazolam when both are taken together. Specifically, researchers tracked how much midazolam and one of its breakdown products (called 1-hydroxy midazolam) appeared in the blood, and how quickly, to understand whether risdiplam changes how the body handles midazolam. The reported data shows that in Part 2, when participants took midazolam on its own, the total amount of midazolam measured in the blood over time (a figure called AUCinf, which captures the full exposure) was 22.6 h·ng/mL. When midazolam was taken alongside risdiplam, that figure was 25.1 h·ng/mL. A similar measure covering only the observed timeframe (AUClast) was 19.9 h·ng/mL for midazolam alone and 22.0 h·ng/mL when combined with risdiplam. The peak blood concentration of midazolam (the highest level recorded) was 7.65 ng/mL alone and 8.96 ng/mL in combination with risdiplam. For the breakdown product 1-hydroxy midazolam, the reported total exposure figures were 8.66 h·ng/mL (alone) versus 9.41 h·ng/mL (with risdiplam) for AUCinf, 7.75 h·ng/mL versus 9.43 h·ng/mL for AUClast, and peak concentrations of 3.18 ng/mL versus 4.10 ng/mL 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.

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

• NCT02644668 · results posted 31 August 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT02644668) looked at a medicine called reldesemtiv in people with spinal muscular atrophy (SMA), a condition that affects muscle strength and movement. A total of 70 people took part — 26 received a placebo (a dummy treatment with no active ingredient), 24 received reldesemtiv at a lower dose (150 mg twice daily), and 20 received reldesemtiv at a higher dose (450 mg twice daily). The trial ran for 8 weeks and measured several things related to breathing and muscle function, including how much air participants could breathe in and out, how hard they could inhale and exhale, overall muscle strength across key muscle groups, and scores on two standard movement and motor function scales. The reported data shows the following changes from the start of the trial to week 8. For lung capacity (called Forced Vital Capacity), all three groups showed a very small decline: −0.02 litres for placebo, −0.03 litres for the lower dose, and −0.07 litres for the higher dose. For maximum inhalation pressure, the reported changes were −2.62, −5.56, and −1.63 cm H₂O respectively. For maximum exhalation pressure, the placebo group showed a change of −4.22 cm H₂O, while the lower-dose group showed +7.47 and the higher-dose group +8.93 cm H₂O. For overall muscle strength (averaged across six body locations and expressed as a percentage change from the starting point), the placebo group showed +14.34%, the lower-dose group +9.76%, and the higher-dose group −0.88%. On the Hammersmith motor function scale (scored 0–66, higher is better), changes were +1.40, +1.02, and +0.39 points respectively. On the upper limb motor function scale (scored 0–43, higher is better), changes were +0.54, +1.15, and +0.43 points. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03306277 · results posted 16 July 2020

  According to the results reported on ClinicalTrials.gov, this trial (NCT03306277) enrolled 22 infants who received a single treatment called onasemnogene abeparvovec-xioi (a gene therapy). The trial was studying children with a serious muscle-weakening condition called spinal muscular atrophy (SMA). The study had two main things it was measuring: whether the children could sit up on their own for at least 30 seconds, and whether they survived without needing permanent breathing support. Nineteen of the 22 participants completed the study, and three did not. The reported data shows that 13 out of 22 participants were recorded as being able to sit independently for at least 30 seconds. For the survival measure — meaning staying alive and not needing round-the-clock breathing assistance — 20 out of 22 participants met that definition. On the two secondary measures, 9 out of 22 participants were recorded as meeting all the criteria for "ability to thrive" at 18 months of age (which included eating without mechanical feeding support, being able to swallow thin liquids safely, and maintaining their weight). Separately, 18 out of 22 participants were reported as not requiring any daily ventilator (breathing machine) support at 18 months of age. It is worth noting that these numbers are simply counts of how many participants met each pre-defined measure at the time points assessed — they do not on their own tell us what outcomes to expect for any individual child, and comparison figures from an untreated group were not included in this data 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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• NCT00568802 · results posted 2 October 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT00568802) enrolled 27 participants in total, all grouped together under a single study arm for tracking purposes. Twenty-four participants completed the study, while three did not finish. The trial was comparing hydroxyurea (a medicine) against a placebo (a dummy treatment with no active ingredient) in participants with spinal muscular atrophy, a condition affecting muscle strength and movement. The study set out to measure two main things: how well participants could perform motor tasks (such as movement tests and timed physical activities), and how often unwanted health events or abnormal laboratory results occurred. Several additional measures were also tracked, including lung function tests, nerve and muscle activity estimates, and levels of specific proteins and genetic material in the body related to the condition. The reported data shows that, unfortunately, no numerical results were submitted to ClinicalTrials.gov for any of the outcome measures — neither the primary outcomes (motor function testing and frequency of adverse events or lab abnormalities) nor the secondary outcomes (lung function, muscle nerve estimates, or biomarker levels). This means the actual figures from the study are not available in the registered results, and it is not possible to describe what the numbers showed for either the hydroxyurea group or 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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• NCT00568698 · results posted 2 October 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT00568698) enrolled 29 participants in total. It was studying hydroxyurea compared to a placebo (an inactive dummy treatment) in what appears to have been a trial involving children with spinal muscular atrophy, a condition affecting muscle strength and movement. The trial aimed to measure both how safe the treatment appeared to be and whether it had any effect on survival and breathing function, as well as certain biological markers in the body. Of the 29 people who started the trial, 18 completed it and 11 did not. The reported data shows that four outcome measures were tracked: the frequency of side effects and abnormal lab results; length of survival and the age at which participants became dependent on a ventilator (a breathing machine); a measure of nerve and muscle activity called Motor Unit Number Estimation; and levels of specific proteins and genetic material in the body linked to the condition. However, the actual numerical results for all four of these outcome measures were not reported in the structured data submitted to ClinicalTrials.gov — the figures simply do not appear in the record available, so it is not possible to describe what the numbers showed for any of these measures. Because no outcome numbers were provided in the submitted data, it is not possible to draw any conclusions from this record about what the trial found regarding either the hydroxyurea or 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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• NCT02628743 · results posted 9 August 2019

  According to the results reported on ClinicalTrials.gov, this trial enrolled 131 participants, all of whom received a medicine called olesoxime. The trial was studying people with spinal muscular atrophy (SMA), a condition that affects muscle strength and movement. The study tracked adverse events (that is, any unwanted medical occurrences during the trial), as well as changes in participants' ability to move, their quality of life, and the levels of olesoxime measured in their blood. Notably, the reported data shows that none of the 131 participants were recorded as having "completed" the trial — all 131 were listed under "not completed," though the data does not explain the reasons for this in full. The reported data shows that 27.5% of participants experienced adverse events (AEs) and 91.6% experienced serious adverse events (SAEs) — these figures describe how many people had unwanted medical occurrences, but the data as reported does not allow a judgement to be made about whether these were caused by the medicine. For the motor function assessments — which used a scoring tool called the Motor Function Measure (MFM), where a higher score means better physical ability — participants started with an average score of around 30% of the maximum for two of the three movement categories combined, and around 41% for all three categories combined. Over the course of the trial, the reported changes in these scores were small decreases (for example, the largest reported change was around minus 4 to minus 5 percentage points). Similarly, quality-of-life scores — rated on a scale where higher means better — started at roughly 55–59 out of 100 and showed small reported changes over time, mostly small decreases of a few points. Blood levels of olesoxime were also measured at various time points, with figures ranging widely across the study period; the data notes that the dose was increased after week 104. 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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• NCT03056144 · results posted 26 July 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT03056144) enrolled only one participant in a single intervention group. The study was measuring several aspects of physical ability and development in a child, including gross motor function, walking capacity, trunk (torso) control in sitting, everyday functional skills (such as self-care, moving around, and social function), as well as height and weight. The reported data shows that results were only available for two of the six outcome measures. For trunk control in sitting — scored on a scale where 8 means full trunk control has been achieved — the single participant scored 8 out of 8. For the everyday functional skills assessment, which covers three areas, the reported data shows scores of 68 out of a possible 73 for self-care, 14 out of a possible 59 for mobility, and 64 out of a possible 65 for social function. No results were reported for the gross motor function test, the 2-minute walking test, height, or weight — the data for those measures was not submitted. It is worth noting that with only one person taking part, these numbers reflect the experience of a single individual and cannot be used to draw broader conclusions. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01839656 · results posted 30 October 2018

  According to the results reported on ClinicalTrials.gov, this trial (NCT01839656) involved 21 babies and young children with spinal muscular atrophy (SMA), a condition that affects muscle strength and movement. Five children received a lower dose of nusinersen (6 mg) and 16 received a higher dose (12 mg). The trial was measuring whether nusinersen, which was given by injection into the fluid around the spine, could lead to improvements in physical movement milestones — things like head control, rolling, sitting, and walking — as well as survival without needing permanent breathing support. The reported data shows that, looking at motor (movement) milestones at the last study visit, 25% of children in the 6 mg group and 68.8% of children in the 12 mg group were recorded as showing improvement. For survival without needing permanent breathing support, the reported figures were 25% in the 6 mg group and 62.5% in the 12 mg group. The reported data also shows that 25% of children in the 6 mg group and 62.5% in the 12 mg group were recorded as having improved scores on a separate movement test (called CHOP-INTEND), which rates a range of physical movements on a scale from 0 to 64. Regarding unwanted medical events, 4 out of 5 children in the 6 mg group and all 16 children in the 12 mg group were reported as experiencing at least one adverse event, while serious adverse events were reported in 3 out of 5 and 13 out of 16 children respectively. It is worth noting that only 2 children in the 6 mg group and none in the 12 mg group completed the full study, meaning the numbers involved are very small and should be kept in that context 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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• NCT01736553 · results posted 4 May 2018

  According to the results reported on ClinicalTrials.gov, this study enrolled 26 infants diagnosed with Spinal Muscular Atrophy (SMA) — a condition affecting the muscles and movement — and 27 healthy infants for comparison. The trial tracked both groups over their first two years of life, measuring how their movement and motor skills developed using several standardised tests, and also looked at biological markers in the blood and nerve signals to see how these differed between the two groups. Notably, only 7 of the 26 SMA infants completed the study, compared with 23 of the 27 healthy infants. The reported data shows the following across the movement tests and biological measures. On the TIMPSI movement test (scored 0–99, where higher means better movement ability), SMA infants scored in the range of roughly 23 to 41 across study visits, while healthy infants scored in the range of roughly 85 to 89. On the CHOP-INTEND test (scored 0–64), which was only used for SMA infants who scored lower on the TIMPSI, scores for those infants ranged from about 7 to 18 across visits. On the AIMS movement test (scored 0–58), used for infants with stronger TIMPSI scores, SMA infants recorded scores roughly between 12 and 22, while healthy infants scored between about 19 and 39 across visits. For the nerve signal test (CMAP, measuring electrical activity in millivolts), SMA infants returned values ranging from roughly 0 to 1.07 mV, compared with roughly 6.0 to 6.7 mV in healthy infants. For the blood-based biological markers, the reported data shows that a gene activity measure (SMN/HPRT ratio) ranged from about 0.47 to 0.66 in SMA infants versus about 1.11 to 1.29 in healthy infants, and a protein level measure ranged from roughly 3,109 to 8,502 units in SMA infants versus roughly 6,636 to 12,030 units in healthy infants across the study visits. 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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• NCT00878436 · results posted 4 December 2017

  According to the results reported on ClinicalTrials.gov, this trial looked at a drug called LBH (panobinostat) given at two different doses — 40 mg and 20 mg — to men with prostate cancer. A total of 52 people took part: 28 in the higher-dose group and 24 in the lower-dose group. The trial's main focus was measuring how many participants remained free of their cancer progressing and did not experience a worsening of symptoms, tracked using blood tests (PSA levels), CT scans, and bone scans. Only those who completed at least two treatment cycles were assessed for this main outcome. The reported data shows two sets of figures for the main outcome (likely measured at different points in time, though the specific timepoints are not clearly labelled in the submitted data). In the first set of results, 24% of participants in the 40 mg group and 9% in the 20 mg group were reported as free of progression without symptomatic deterioration. In the second set, those figures were 42% in the 40 mg group and 19% in the 20 mg group. Regarding how long it took for PSA levels to rise (a sign of progression), and how many patients achieved a 50% or greater drop in PSA by nine months, the reported data shows no figures were submitted for either of these secondary outcomes — in one case the data was described as unavailable, and in the other no measurements were recorded. 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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• NCT02024932 · results posted 11 August 2017

  According to the results reported on ClinicalTrials.gov, this trial tested an investigational drug called BVS857 and involved a total of 37 people across several groups. The trial was split into two parts (Part A and Part B) and included both open-label groups (where everyone knew what was being given) and double-blind groups (where neither the participants nor the medical team knew who received the drug or a dummy treatment called a placebo). The trial was primarily looking at how many people experienced unwanted health events (called adverse events) while taking the drug, and in Part B it also measured changes in thigh muscle size using MRI scans. The reported data shows that in terms of adverse events — unwanted health events that occurred during the study — nearly all participants across all groups experienced at least one. For example, in the largest group (BVS857 Part B Double Blind, 18 participants), 17 reported at least one adverse event, while 8 out of 9 placebo participants did the same. No serious adverse events or deaths were reported in any group. Regarding the severity of those events, the reported data shows a mix of mild and moderate adverse events across groups, with no severe adverse events reported for most groups. For the main measurement of thigh muscle volume change in Part B, the BVS857 double-blind group showed an average change of 0.0% from the starting point, while the placebo group showed an average change of −3.4%. For lean body mass, the BVS857 group showed an average increase of 0.77 kilograms compared to 0.16 kilograms in the placebo group. On a physical function and muscle endurance scoring tool (rated 0–45, where higher is better), the BVS857 group showed an average improvement of 1.0 points, while the placebo group showed an average improvement of 2.3 points. It is worth noting that the groups in this trial were very small, which limits how much can be read into these numbers. Some participants did not complete the trial, and not all outcome data was reported for every 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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• NCT02193074 · results posted 28 July 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT02193074) enrolled 121 infants in total — 41 in the control group and 80 in the nusinersen group. The trial was measuring things like whether children showed improvements in physical movement milestones (such as head control, sitting, or rolling), how long it took before children either died or needed permanent breathing support, and changes in muscle function tests. The study was stopped early, with 24 control participants and 65 nusinersen participants recorded as having completed the trial (including those who completed due to early termination). The reported data shows that for the main ("primary") outcome of motor milestone improvement, 0% of children in the control group met the criteria for being a "responder" (meaning they showed meaningful gains in movement categories), compared with 51% of children in the nusinersen group. For the other primary outcome — the proportion of children who died or needed permanent breathing support over time — the reported data shows that at the final measured point, approximately 73.5% of the control group and 44.7% of the nusinersen group had reached that outcome. For the secondary outcomes, 3% of control participants and 71% of nusinersen participants showed a meaningful improvement on a separate muscle movement test (CHOP-INTEND). Regarding permanent ventilation, 68% of the control group and 77% of the nusinersen group did not require it. In a nerve-signal test (CMAP), 5% of the control group and 36% of the nusinersen group met the responder criteria. 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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• NCT02294461 · results posted 12 April 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT02294461) enrolled 395 men with prostate cancer — 202 received enzalutamide and 193 received a placebo (an inactive dummy treatment) during the main double-blind phase, where neither participants nor researchers knew who was getting which treatment. A further 51 participants who had originally been in the placebo group later crossed over to receive enzalutamide in an open-label phase (where everyone knew what was being given). The trial was primarily measuring how long it took for a protein in the blood called PSA — which is linked to prostate cancer activity — to show signs of rising again (called "PSA progression"). It also measured a range of other things, including how long participants lived overall, how long before cancer showed visible growth on scans, and how long before certain bone complications or chemotherapy were needed. The reported data shows that, for the primary measure, the median time until PSA progression was 8.31 months in the enzalutamide group compared with 2.86 months in the placebo group. (Median means the point at which half the participants in each group had reached that milestone.) For overall survival, the reported median was 39.06 months in the enzalutamide group and 27.10 months in the placebo group. For the measure of how long before cancer visibly progressed on scans (radiographic progression-free survival), a median figure was not able to be calculated for the enzalutamide group — meaning not enough participants in that group had reached that point during the study period — while the placebo group's median was 5.29 months. Similarly, for time to first bone complication, no median figure was reported for either group. For time to starting chemotherapy, no median was reached in the enzalutamide group, while the placebo group's median was 13.93 months. Regarding PSA response (a drop of 50% or more from the starting level), the reported data shows this was seen in 120 out of 202 participants in the enzalutamide group, compared with 15 out of 193 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.

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• NCT01703988 · results posted 13 March 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT01703988) tested four different doses of a medicine called nusinersen — 3 mg, 6 mg, 9 mg, and 12 mg. A total of 34 people took part across the four dose groups (8, 8, 9, and 9 participants respectively). The trial was primarily measuring unwanted medical events (called adverse events) that occurred during the study, and secondarily measuring how the drug moved through the body — including how much of it appeared in the blood and the fluid around the brain and spinal cord (called cerebrospinal fluid). The reported data shows that when it came to the primary outcome — tracking unwanted medical events — all 8 participants in the 3 mg group, 6 out of 8 in the 6 mg group, all 9 in the 9 mg group, and all 9 in the 12 mg group experienced at least one such event. No participants in any group were reported to have experienced a serious adverse event (one that was life-threatening, required hospitalisation, or met similar criteria). A small number of participants — 2 in the 3 mg group and 1 in the 12 mg group — discontinued the study due to an adverse event. For the secondary outcomes tracking how the drug behaved in the body, the reported data shows that the peak level of nusinersen detected in the blood rose with each higher dose, ranging from around 51.5 ng/mL at 3 mg up to around 208 ng/mL at 12 mg. The time it took to reach that peak level in the blood was broadly similar across all groups, sitting roughly between 4 and 6 hours. Drug levels were also detected in the cerebrospinal fluid, with figures varying across doses and time points. Kidney clearance data (how the body removed the drug through urine) was only collected for the 12 mg group, as that was specified in the study plan. 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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• NCT01402570 · results posted 17 February 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT01402570) enrolled 20 participants in a single study group, of whom 19 completed the trial and 1 did not finish. The trial was measuring four things across multiple time points: participants' self-reported physical functioning and fatigue (using a standardised questionnaire system called PROMIS, where scores are compared against a general population average of 50), how efficiently participants were sleeping (the proportion of time in bed actually spent asleep, recorded in sleep diaries), and how many steps per day participants were taking (tracked using an arm-worn activity monitor). The reported data shows the following numbers across the measurement time points. For physical functioning (where higher scores mean better function, and 50 is the population average), scores were reported as 37.8, 37.9, 37.5, and 38.1. For fatigue (where higher scores mean greater fatigue), scores were reported as 56.0, 54.9, 53.4, and 52.8. For sleep efficiency (where 100% would mean all time in bed was spent sleeping), the reported figures were 82.1%, 86.9%, 87.6%, and 87.8%. For daily steps, the reported counts were 4,004, 3,591, 3,599, and 3,693 steps per day. The data as submitted does not label which time point each individual number belongs to, so the exact timing of each measurement cannot be confirmed from the available information. 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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• NCT01393444 · results posted 12 December 2016

  According to the results reported on ClinicalTrials.gov, this trial enrolled 6 participants, all listed under the group "Direct Brain Interface Users." The trial was looking at whether people could control external devices — such as computer cursors, virtual reality environments, and assistive devices like hand braces or muscle stimulators — using their brain activity recorded through a system called ECoG (electrocorticography), which involves sensors that pick up electrical signals from the surface of the brain. Of the 6 people who started the trial, 3 completed it and 3 did not complete it; the reasons for not completing were not detailed in the reported data. The reported data shows that, for the primary outcome — measuring how many participants could successfully control a variety of external devices using their recorded brain signals — the number reported was 3 out of 6 participants. For the secondary outcome — measuring how many participants could achieve direct brain control of assistive devices specifically using the ECoG-based system — the number reported was also 3 out of 6 participants. No further breakdown of how well or how consistently control was achieved 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.

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• NCT00528268 · results posted 3 July 2015

  According to the results reported on ClinicalTrials.gov, this trial looked at sodium phenylbutyrate in babies and very young children with spinal muscular atrophy (SMA), a condition that affects muscle strength and movement. There were two groups in the trial — Cohort 1 and Cohort 2. Six children started in Cohort 1 (five completed the trial, one did not), and eight children started in Cohort 2 (seven completed, one did not). In total, 14 children began the trial across both groups. The reported data shows that the primary outcome — what the trial was mainly set up to measure — looked at how many children were able to reach certain physical development milestones during the study, such as being able to sit without support or walk on their own. According to the results reported on ClinicalTrials.gov, in Cohort 1, one child reached the first milestone and no children reached the second milestone. In Cohort 2, eight children reached the first milestone and one child reached the second milestone. Please note that the data as submitted does not provide labels distinguishing exactly which milestone each set of numbers refers to, so a full breakdown cannot be described here. It is worth noting that, despite the trial's title referring to "safety and tolerability" (meaning whether the treatment caused any problems and whether it was manageable to take), the specific safety and tolerability numbers — such as any side effects recorded — were not included in the structured results data submitted to ClinicalTrials.gov, so those figures cannot be described 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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• NCT00661453 · results posted 15 June 2015

  According to the results reported on ClinicalTrials.gov, this trial enrolled 38 children with Spinal Muscular Atrophy (SMA) Type 1, which is a serious genetic condition affecting muscle strength. Of the 38 who started the trial, 25 completed it and 13 did not finish. The trial was measuring things like body weight and body composition (how much fat and lean tissue a child has), as well as other markers of nutritional health. It also aimed to track things like time to death or the need for a breathing machine, and assessments of movement and function. The reported data shows that for the main nutritional measurement — which tracked body weight in grams at different points — the figures reported were approximately 3,011 g, 3,618 g, 4,317 g, 4,316 g, 4,994 g, and 5,134 g across various time points. These appear to represent recorded weight measurements during the study. For several other outcomes that were listed — including the laboratory safety data, time to death or ventilator dependence, caregiver-rated function scores, movement assessments, and genetic marker measurements — no numerical results were reported in the data submitted to ClinicalTrials.gov. Because a number of the planned outcome measures did not have results included in the submission, the picture from this trial is incomplete based on what is publicly available. The data was not reported for those measures, so no conclusions can be drawn from them 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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• NCT00918385 · results posted 29 October 2014

  According to the results reported on ClinicalTrials.gov, this trial enrolled 31 men in total — 14 in a group called "High AR Nilutamide" (Arm 1) and 17 in a group called "Low AR Dasatinib" (Arm 2). The trial was testing whether a biomarker called AR (androgen receptor) activity could be used to match patients to one of two different cancer medicines — nilutamide or dasatinib — and then later combining both medicines. The trial measured how long participants went without their disease getting worse (called "progression-free survival"), as well as how many participants had their tumours shrink or disappear during treatment. The reported data shows that for the primary outcome — time from the start of single-drug treatment until the disease progressed or death occurred — the median figure (the midpoint value across participants) was 2.8 months for Arm 1 (nilutamide) and 2.6 months for Arm 2 (dasatinib). For the secondary outcomes, the reported data shows that 0% of participants in either group had a complete response (tumour fully disappearing) or a partial response (tumour shrinking by at least 30%) based on standard imaging criteria known as RECIST. Regarding how many participants completed each phase: in the single-drug phase, 12 of 14 in Arm 1 and 11 of 17 in Arm 2 completed it; in the combination phase, 6 of 12 in Arm 1 and 3 of 11 in Arm 2 completed it. No reasons for non-completion were 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.

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• NCT01233817 · results posted 12 March 2013

  According to the results reported on ClinicalTrials.gov, this trial (NCT01233817) enrolled 12 people who had been diagnosed with Spinal Muscular Atrophy (SMA), a condition that affects the muscles. Nine participants completed the trial, while three did not finish. The trial was measuring muscle strength using a technique called Quantitative Muscle Analysis (QMA), which involves participants pushing or pulling against a fixed point so that the force their muscles can produce can be measured in kilograms. The reported data shows that the single outcome measure recorded was a muscle strength value of 0.39 kilograms. This figure was reported for the group as a whole. It is worth noting that no secondary outcome measures, comparison groups, or additional detail about how this number changed over the course of the trial were included in the data submitted to ClinicalTrials.gov, so those figures cannot be described 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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• NCT00365599 · results posted 26 March 2012

  According to the results reported on ClinicalTrials.gov, this trial enrolled 43 participants, all of whom completed the study. Every participant received a combination of two medicines: vorinostat and tamoxifen. The trial was measuring how many participants showed an "objective response" — meaning their tumours shrank or disappeared by a measurable amount based on scan measurements — as well as how long it took for the disease to progress, and how many participants experienced serious unwanted health events. The reported data shows that out of 43 participants, 8 showed an objective response to the treatment combination. For the secondary outcomes, the reported median time to progression — that is, the midpoint figure for how long it took, across participants, before the disease started getting worse again — was 10.3 months. The reported data also shows that 4 out of 43 participants experienced what were classified as serious adverse events (that is, significant unwanted health occurrences that were formally recorded and reported). It is worth noting that this trial had only one group, so there was no separate comparison group receiving a different or dummy treatment, which means the reported numbers reflect only the experience of participants who received this particular combination. 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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• NCT00227266 · results posted 3 May 2011

  According to the results reported on ClinicalTrials.gov, this trial enrolled 94 participants across three groups. The first group ("Cohort 1a") had 31 participants who started on a placebo and then switched to the treatment — these were children described as "sitters" (meaning they could sit but not stand or walk independently). The second group ("Cohort 1b") had 30 participants who were also sitters and received the treatment throughout. The third group ("Cohort 2") had 33 participants who were "standers and walkers." The trial was measuring two main things: safety through regular blood tests monitoring liver function and appropriate dosing, and motor function (movement ability). It also looked at several secondary measures, including a nerve-and-muscle electrical signal test called a CMAP (Compound Motor Action Potential), levels of a specific protein in the blood, and quality-of-life questionnaires completed by parents and children. The reported data shows that for the primary outcomes — the safety lab results and the motor function assessments — no numerical results were submitted to ClinicalTrials.gov, so those figures are not available to describe here. For the secondary CMAP nerve signal measurements, the reported data shows average (mean) readings at two time points for each group. In Cohort 1a (placebo then treatment), the mean CMAP was 2.28 millivolts (mV) at the first time point and 2.32 mV at the second. In Cohort 1b (sitters on treatment), it was 2.93 mV then 2.37 mV. In Cohort 2 (standers and walkers), it was 5.52 mV then 6.56 mV. Similarly, the median (middle value) CMAP readings were: Cohort 1a — 1.91 mV then 1.44 mV; Cohort 1b — 2.20 mV then 1.80 mV; and Cohort 2 — 5.30 mV then 5.85 mV. The results for the quality-of-life questionnaires and the protein blood sample measurements 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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• NCT00439218 · results posted 20 July 2010

  According to the results reported on ClinicalTrials.gov, this trial enrolled a total of five participants across two cohorts. Both cohorts received the same dose level of 500 mg/kg/day of the study treatment. Four participants started in Cohort 1, with three completing the trial and one not completing it. One participant started and completed Cohort 2. The trial was measuring whether the treatment caused any serious side effects (called "dose limiting toxicities") at this dose level, and whether it changed the levels of a specific protein and a related molecule (called SMN protein and SMN mRNA) in participants' blood. These blood markers are relevant to a condition called spinal muscular atrophy. The reported data shows that no numerical results were submitted to ClinicalTrials.gov for any of the outcome measures. This means that the specific numbers for serious side effects, SMN protein levels, SMN mRNA levels, drug safety monitoring, or the pharmacokinetic measurements — which track how the drug moves through the body, including its peak concentration in the blood and how long it takes to reach that peak — were not reported in the structured results data. Because these figures are absent from the submitted data, it is not possible to describe what the measurements found. 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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• NCT00303446 · results posted 22 June 2010

  According to the results reported on ClinicalTrials.gov, this trial enrolled 50 people in total — 25 received a placebo (a dummy treatment with no active ingredient) and 25 received a medicine called dutasteride. By the end of the two-year study, 23 people in the placebo group and 21 in the dutasteride group had completed it. The trial was looking at whether dutasteride had any effect on muscle strength and physical function in participants with a muscle condition, with the main measurement being changes in overall muscle strength compared to the start of the trial. The reported data shows that for the primary (main) outcome — muscle strength measured across 22 muscle groups and expressed as a percentage change from the starting point — the placebo group showed a change of −2.2% at 12 months and −4.5% at 24 months, while the dutasteride group showed a change of +3.1% at 12 months and +1.3% at 24 months. For the secondary (additional) outcomes, the reported data shows mixed figures across both groups. On a hands-on muscle strength rating scale (where 5 is best), changes from the starting point were small in both groups at both time points. On a physical function and endurance score (where 45 is best), both groups showed small negative changes — meaning slight decreases from baseline — at 12 and 24 months. For the two-minute walking test, the placebo group's average distance changed by +8.1 metres at 12 months and +2.2 metres at 24 months, while the dutasteride group's changed by −0.8 metres and −1.6 metres respectively. A blood marker called creatine kinase (an enzyme sometimes associated with muscle activity) also changed in both groups across the two time points. Swallowing assessments showed small changes in both groups, with the placebo group showing slightly more negative shifts at both time points. It is worth noting that the reported data does not include information about whether the differences between the two groups were considered statistically meaningful (that is, unlikely to be due to chance), so the numbers alone cannot tell us how significant these differences may or may not be. 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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• NCT00439569 · results posted 12 February 2010

  According to the results reported on ClinicalTrials.gov, this trial enrolled a total of nine children across two groups (called cohorts). Seven children started in Cohort 1 and two started in Cohort 2 — both cohorts were given the same dose of the study drug (500 mg/kg/day). The trial was investigating a treatment for Spinal Muscular Atrophy (SMA), a condition that affects muscle strength and movement. The study was looking at how the body handled the drug, whether certain side effects occurred at that dose level, and whether the drug had any effect on two biological markers in the blood — a molecule called SMN mRNA and a protein called SMN protein — both of which are linked to the condition. The reported data shows that no numerical results were submitted to ClinicalTrials.gov for any of the outcome measures. This means that the specific numbers for side effect thresholds (called "dose limiting toxicities"), changes in SMN mRNA levels, changes in SMN protein levels, general adverse events (unwanted health events), and drug levels in the blood (including peak concentration and the time it took to reach that peak) were all not reported in the structured results data. Of the nine children who started the trial, only four completed it — three from Cohort 1 and one from Cohort 2 — but the reasons for non-completion and any related findings were not included in the submitted data. Because no outcome numbers were provided in the ClinicalTrials.gov submission, it is not possible to describe what the trial found in terms of measurable results. The reported data shows only that the trial took place and what it intended to measure, but the actual figures are absent from the public record. 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.