Reported trial results for Huntington's Disease

24 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

• NCT05655520 · results posted 17 December 2025

  According to the results reported on ClinicalTrials.gov, this trial enrolled 153 participants across two groups: 72 people in "Cohort 1 (Direct Rollover)" and 81 people in "Cohort 2 (Gap Rollover)." These labels suggest participants had previously been in a related study and either moved straight into this one or had a break in between. The trial's primary focus was on tracking unwanted medical events (called treatment-emergent adverse events, or TEAEs — meaning any new or worsening health issue that appeared after starting the study treatment) and on measuring changes in basic physical signs like blood pressure and heart rate. The reported data shows that in Cohort 1, 46 out of 72 participants experienced at least one such unwanted medical event, and the same number — 46 out of 81 — did so in Cohort 2. Breaking those down by how serious they felt: in Cohort 1, 21 participants had mild events, 22 had moderate events, and 3 had severe events; in Cohort 2, 24 had mild, 18 had moderate, and 4 had severe events. A small number of participants left the study because of these events — 2 in Cohort 1 and 3 in Cohort 2. For the physical measurements, the reported changes from the start of the study to the last recorded point were very small: blood pressure shifted by only a few millimetres of mercury (mmHg) in either direction depending on position and group, heart rate changed by roughly 2 beats per minute across both groups, and breathing rate was essentially unchanged (0.0 to −0.1 breaths per minute). It is worth noting that very few participants formally "completed" the study as defined by the trial — only 2 in Cohort 1 and 4 in Cohort 2 — though the data does not explain why the rest did not reach that milestone. No secondary outcome data appears to have been reported in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05107128 · results posted 5 September 2025

  According to the results reported on ClinicalTrials.gov, this trial (NCT05107128) looked at a drug called SAGE-718 in people with Huntington's disease. A total of 189 people took part — 94 received a placebo (a dummy treatment with no active ingredient) and 95 received SAGE-718. The trial's main focus was on whether SAGE-718 had any effect on a test called the Symbol Digit Modalities Test (SDMT), which measures thinking speed and attention by asking people to match symbols to numbers within 90 seconds. The trial also measured several other things, including daily independence, motor timing, problem-solving, and how much difficulty people experienced with everyday tasks at home. The reported data shows that on the primary SDMT test, both groups showed a small increase in score from where they started: the placebo group's average score went up by 2.0 points, while the SAGE-718 group's average score went up by 0.8 points (out of a possible 110). For the secondary measures, the reported data shows that on the independence scale, both groups showed a small decline (placebo: −0.8 points; SAGE-718: −1.5 points). On a timed task-switching test (Trail Making Test Part B), both groups were faster than at the start, with the placebo group taking 5.7 seconds less and the SAGE-718 group taking 10.7 seconds less. On a computerised problem-solving test, both groups showed a small improvement in response time (placebo: −4.56 seconds; SAGE-718: −3.85 seconds). On a finger-tapping timing test, the placebo group showed a small improvement (0.710 units) while the SAGE-718 group showed a small decline (−0.157 units). On a self-reported scale of difficulty with everyday tasks at home, the placebo group reported a larger improvement (−4.6 points) compared with the SAGE-718 group (−0.7 points). These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05032196 · results posted 12 August 2025

  According to the results reported on ClinicalTrials.gov, this trial tested an investigational drug called WVE-003, which is being studied in the context of Huntington's disease. The trial was set up in two parts: a single-dose phase, where participants received one dose at one of three different dose levels (30 mg, 60 mg, or 90 mg) or a placebo (an inactive substance); and a multiple-dose phase, where participants received repeated doses of 30 mg or a placebo. In total, 47 people took part in the single-dose phase and 23 people took part in the multiple-dose phase. The main thing the trial was measuring was the number of participants who experienced adverse events (unwanted or unexpected health changes) that appeared to be related to the study drug. The reported data shows that in the single-dose phase, 2 out of 16 placebo participants, 1 out of 12 in the 30 mg group, 3 out of 11 in the 60 mg group, and 3 out of 8 in the 90 mg group had adverse events considered related to the study drug. In the multiple-dose phase, none of the 7 placebo participants had such events, while 8 out of 16 participants in the 30 mg WVE-003 group did. The trial also measured how much of the drug appeared in the blood and in the cerebrospinal fluid (the fluid surrounding the brain and spine). The reported data shows that drug levels in the blood and cerebrospinal fluid generally increased with higher doses in the single-dose phase, and that the drug was detectable in the cerebrospinal fluid across all dose groups tested. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT05111249 · results posted 4 November 2024

  According to the results reported on ClinicalTrials.gov, this trial (NCT05111249) involved 26 people in total — 5 received a placebo (a dummy treatment) and 21 received a medicine called branaplam at a dose of 56 mg. The trial was looking at two main things: whether branaplam changed the level of a harmful protein called mutant huntingtin (mHTT) in the fluid surrounding the brain and spine, and how many participants experienced unwanted health events (called adverse events) during the study. It also tracked changes in brain volume using MRI scans over the course of the study. The reported data shows that, by week 17, the level of the mHTT protein in the spinal fluid had changed by an average of about −1.4% in the placebo group and about −26.6% in the branaplam group — meaning both groups showed a reduction, but a larger numerical reduction was recorded in the branaplam group. On the question of unwanted health events, the reported data shows that 2 out of 5 placebo participants and 18 out of 21 branaplam participants experienced at least one adverse event. Serious adverse events were recorded in 1 placebo participant and 14 branaplam participants. For brain volume measured by MRI, the reported data shows small percentage decreases in total brain volume in both groups across the study period, with the branaplam group showing somewhat larger reductions at each time point. The fluid-filled spaces inside the brain (lateral ventricles) were reported to have increased in volume in both groups, with larger percentage increases recorded in the branaplam 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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• NCT03761849 · results posted 7 November 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03761849) involved people with Huntington's disease and tested a medicine called tovorafenib (also referred to as "Tomi") as well as an earlier investigational medicine called RO7234292, compared against placebo (a dummy treatment with no active ingredient). In total, 899 people were enrolled across six groups. Three of those groups — all receiving either RO7234292 or its placebo — did not complete the study as a group (0 completions recorded), while in the tovorafenib and its placebo groups, between 207 and 216 out of roughly 263–264 participants in each group completed the trial. The trial primarily measured changes in a combined Huntington's disease rating scale (called the cUHDRS, which bundles together scores for physical function, movement, and two thinking tasks into a single number) and a separate measure of day-to-day functioning called the Total Functional Capacity (TFC) score. The reported data shows that for the main combined rating scale (cUHDRS), scores changed from the starting point (baseline) as follows: the placebo group in the tovorafenib part of the trial showed a change of −0.630, the tovorafenib every-8-weeks group showed −0.793, and the tovorafenib every-16-weeks group showed −1.173. For context, the trial description notes that a worsening of 1.2 or more on this scale is considered a meaningful decline. For the TFC functioning score (where lower is worse), the reported changes from baseline were −0.883 for the placebo group, −0.921 for the tovorafenib every-16-weeks group, and −1.284 for the tovorafenib every-8-weeks group. Secondary measures — including motor (movement) scores, two thinking/cognitive tests, and a clinician-rated severity scale — were also reported across the groups, with changes in various directions across all groups; the full numbers for each group are recorded on ClinicalTrials.gov. For the clinician severity scale, data was only reported for the tovorafenib and its placebo groups, and was not available for the RO7234292 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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• NCT03515213 · results posted 28 September 2023

  According to the results reported on ClinicalTrials.gov, this trial enrolled 10 people in total — 9 in the active treatment group and 1 in the placebo group. All 10 participants completed the trial with no dropouts. The trial was investigating a treatment for Huntington's disease, and its main focus was on measuring changes in the levels of a specific protein and its genetic instructions (called PGC-1alpha) in participants' bodies over 6 months. Several secondary measures were also tracked, including a blood level of the study drug, as well as scores on standard Huntington's disease rating scales for movement, thinking, and behaviour. The reported data shows the following for the two primary measures, expressed as "log 2 fold change" (a way of describing how much a measurement went up or down relative to a starting point, where 0 means no change): for PGC-1alpha genetic instructions (RNA), the active group showed values of approximately −0.02 at one timepoint and +0.07 at another, while the single placebo participant showed values of +0.32 and +0.48. For PGC-1alpha protein levels, the active group showed values of +0.26 and +0.02, while the placebo participant showed −0.02 and +0.32. For the secondary measure of the drug's blood level (fenofibric acid), the active group showed values of 64, 9,037, and 10,905 ng/ml across three timepoints, compared with 5, 10, and 19 ng/ml in the placebo participant. On the movement rating scale (where higher scores mean greater difficulty), the active group averaged 19.56 then 21.22, versus 34 then 27 for the placebo participant. On the thinking assessment (where higher scores mean less difficulty), the active group averaged around 24–26 across timepoints, compared with 18–22 for the placebo participant. Behavioural scale scores were broadly similar between groups across all timepoints. It is important to note that with only 10 participants — including just one in the placebo group — the reported data shows a very small snapshot, and no firm conclusions can be drawn from numbers of this size. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03842969 · results posted 31 August 2023

  According to the results reported on ClinicalTrials.gov, this trial (NCT03842969) looked at a drug called tominersen, given at different dosing schedules to people with Huntington's disease. A total of 236 participants took part across three groups: 17 received the drug every 4 weeks (Q4W), 138 received it every 8 weeks (Q8W), and 81 received it every 16 weeks (Q16W). The trial was measuring things related to safety and tolerability — including unwanted health events that occurred during treatment, any signs of suicidal thoughts or behaviours, and changes in thinking and memory using a standard assessment tool. Notably, none of the participants were recorded as having "completed" the study, with all participants listed under "not completed." The reported data shows that the percentage of participants who experienced unwanted health events (called treatment-emergent adverse events — meaning health issues that appeared or worsened after starting the drug) was 71.4% in the every-4-weeks group, 83.9% in the every-8-weeks group, and 81.8% in the every-16-weeks group. For suicidal thoughts or behaviours, as measured by a standardised questionnaire, the reported data shows that 1 participant in the every-4-weeks group, 16 in the every-8-weeks group, and 3 in the every-16-weeks group recorded some form of suicidal ideation or behaviour during the study. Regarding thinking and memory (measured on a scale from 0 to 30, where lower scores mean greater difficulty), starting scores across the groups ranged roughly from about 19.75 to 25.33. Change-from-baseline figures were only partially reported — the every-4-weeks group showed a change of −4.00 points, while some subgroups in the every-8-weeks group showed small positive changes of around 0.16 to 0.46 points; change data for several other subgroups was not reported in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT03225846 · results posted 25 April 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT03225846) enrolled 88 people in total across seven groups. Twenty-two participants received a pooled placebo (a dummy treatment), while the remaining 66 received one of six different doses of an investigational drug called WVE-120102, ranging from 2 mg up to 32 mg. The trial was primarily set up to track the number of participants who experienced unwanted health events (called "adverse events") during the study, including those that were severe or serious. It also measured how the drug moved through the body — for example, how high the drug's level in the blood got, and how quickly that peak level was reached. The reported data shows that in the primary outcomes, most participants across all groups — including those on placebo — experienced at least one adverse event during the trial. For example, 20 out of 22 placebo participants had an adverse event, compared to numbers ranging from 4 to 13 out of each active-dose group. Regarding severe adverse events, the highest-dose group (32 mg) had 9 out of 13 participants affected, compared to 2 out of 22 in the placebo group. Serious adverse events (those involving hospitalisation or other significant consequences) were reported for 9 participants in the 32 mg group, compared to none in the placebo group. Six participants in the 32 mg group also withdrew from the trial due to adverse events, while no withdrawals for this reason were reported in any other group. The reported data for the secondary outcomes shows that as the dose increased, the peak level of WVE-120102 measured in the blood generally rose — from approximately 1.35 ng/mL at the 2 mg dose up to 96.21 ng/mL at the 16 mg dose (figures for the 32 mg dose were not reported in the submitted data). The time taken to reach that peak level generally ranged from roughly 1.2 to 3.3 hours across the different dose 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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• NCT03225833 · results posted 10 February 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT03225833) enrolled 61 people in total across six groups: 16 received a placebo (an inactive substance used for comparison), and the remaining 45 received one of five different doses of an experimental drug called WVE-120101 — either 2 mg, 4 mg, 8 mg, 16 mg, or 32 mg. The trial was primarily measuring how many participants experienced unwanted medical events (called adverse events) after receiving the drug or placebo, and also tracked how the drug moved through the body over time. The reported data shows that when it came to any adverse event during the study, 12 of the 16 placebo participants experienced at least one, compared to 8, 8, 9, 7, and 9 participants in the five WVE-120101 dose groups respectively. For serious adverse events — defined as those involving hospitalisation, life-threatening situations, or lasting disability — none occurred in the placebo group, while 2, 1, 0, 0, and 4 participants experienced them across the five dose groups from lowest to highest dose. Regarding severe adverse events specifically, the numbers reported were 1 in the placebo group and 2, 1, 1, 0, and 5 across the dose groups. A total of 0 placebo participants withdrew from the study due to adverse events, compared to 1, 2, 0, 0, and 2 participants in the WVE-120101 groups. The reported data also shows measurements of how much of the drug appeared in the bloodstream and how quickly. The peak blood concentration of WVE-120101 ranged from approximately 7.70 ng/mL (nanograms per millilitre, a measure of drug level in the blood) in the lowest dose group up to 229.01 ng/mL in the highest dose group. The time it took to reach that peak concentration ranged from about 1.34 hours in the lowest dose group to 4.61 hours in the highest dose group. Some additional measurements for repeated dosing were partially reported, but full data for all groups across all time points was not reported in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01306929 · results posted 9 February 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT01306929) enrolled 134 people with Huntington's Disease, all of whom received the study drug pridopidine. The trial was measuring two main things: how many participants experienced at least one unwanted or unexpected health event (called an adverse event) during the study, and how participants' movement and motor abilities changed over time, as measured by a standard Huntington's Disease rating tool. Of the 134 people who started, 40 completed the study, while 94 did not complete it — though the reasons for this were not detailed in the data provided. The reported data shows that out of 134 participants, 119 experienced at least one adverse event during the trial. Regarding movement and motor abilities, the trial used a scoring tool called the Unified Huntington's Disease Rating Scale Total Motor Score (TMS). On this scale, a score of 0 means no impairment and 124 means the most severe impairment. The reported data shows a series of average scores across different points in time during the study: 38.3, 41.5, 41.7, 44.7, 45.3, 45.7, and 50.0. However, the specific time points these scores correspond to were not clearly labelled in the data provided, so it is not possible to say exactly when each score was 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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• NCT04617860 · results posted 9 February 2022

  According to the results reported on ClinicalTrials.gov, this trial tested a drug called WVE-120102 in 36 people across three starting dose groups — 8 people began on 4 mg, 10 on 8 mg, and 18 on 16 mg. During the trial, many participants had their doses increased over time, with some eventually receiving 32 mg. The trial was measuring safety and tolerability — in other words, it was tracking what kinds of unwanted health events occurred while people were taking the drug and at what doses. Notably, none of the 36 participants completed the trial; all either withdrew or did not finish for other reasons. The reported data shows that across all dose groups, a number of participants experienced what are called "treatment-emergent adverse events" (TEAEs) — these are unwanted health events that occurred during the treatment period. In the 4 mg group, 7 out of 8 participants had at least one such event; in the 8 mg group, 14 participants; in the 16 mg group, 34 participants; and in the 32 mg group, 17 participants. The reported data also shows that more severe events were recorded at higher doses — no participants in the 4 mg or 8 mg groups had a severe event, while 2 in the 16 mg group and 5 in the 32 mg group did. Serious adverse events (a specific medical category for events requiring significant attention) were reported in 0, 1, 5, and 3 participants across the four dose groups respectively. A total of 4 participants withdrew from the trial due to these events (0, 1, 3, and 0 across the dose 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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• NCT04617847 · results posted 9 February 2022

  According to the results reported on ClinicalTrials.gov, this trial (NCT04617847) tested a medicine called WVE-120101 across different doses — 4 mg, 16 mg, and 32 mg. A total of 27 people started the trial (3 in the 4 mg group and 24 in the 16 mg group). Some participants later had their dose adjusted upward during the study. No participants were recorded as having completed the trial in the formal sense, and all were listed under "not completed," though this may reflect how the trial was structured or ended rather than dropouts alone. The reported data shows that the primary focus of this trial was on tracking adverse events — that is, unwanted medical occurrences that happened during treatment. In the 4 mg group, 2 out of 3 participants experienced a treatment-related adverse event, with none classified as severe or serious. In the 16 mg group, 17 out of 24 participants experienced a treatment-related adverse event, with 5 of those classified as severe and 3 classified as serious; 1 person in this group withdrew from the trial due to such an event. In the 32 mg group (which included participants who had their dose increased), 2 out of a small number experienced a treatment-related adverse event, with 1 classified as serious and none classified as severe; no one in this group withdrew due to an adverse event. The reported data shows only these adverse event counts — no other outcome measures, such as measures of how the disease responded, were included in the submitted results. The size of the 32 mg group was not clearly stated in the data as submitted, so a precise participant count for that group cannot be confirmed 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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• NCT02494778 · results posted 17 September 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT02494778) enrolled 248 people who received pridopidine at a dose of 45 mg twice daily. Of those who started, 27 completed the study and 221 did not complete it. The trial ran for up to 106 weeks and was measuring, among other things, any unwanted or unexpected health events (called adverse events) that participants experienced, as well as changes in certain motor (movement) functions measured using specialised sensors on the hands. The reported data shows that out of 248 participants, 193 experienced at least one adverse event over the course of the study. It is important to note that this number alone does not indicate whether those events were serious or minor — only that they were recorded and reported. For the movement measurements, the reported data shows a change from baseline (the starting point) of 0.0 and −0.1 seconds for one hand movement measure (how consistently hand rotation movements started), and changes of −2.9% and −5.9% for another measure (relating to the peak force of hand rotation movements). In these scales, a positive number would indicate worsening, so the reported figures suggest little to no change or a slight movement in the other direction, though the data does not include enough detail to draw firm conclusions. It is worth noting that the data as submitted does not include a comparison group (such as a placebo group), which limits what can be understood from these numbers on their own. Some figures, such as the timepoints for the two measurements in each motor outcome, were not clearly labelled 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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• NCT02006472 · results posted 19 July 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT02006472) tested four different doses of a drug called pridopidine — 45 mg, 67.5 mg, 90 mg, and 112.5 mg (each taken twice daily) — against a placebo (inactive treatment) in people with Huntington's disease. Around 408 people were enrolled across the five groups at the start of the trial, with roughly 81–82 people in each group. The trial ran in two periods: the first up to 26 weeks, and the second continuing to 52 weeks. The main thing being measured was a standardised motor (movement) score called the UHDRS Total Motor Score, which rates things like eye movement, speech, involuntary movements, balance, and gait on a scale of 0 to 124 — where a lower score means better movement ability. The reported data shows that at 26 weeks, all five groups had lower (improved) motor scores compared to where they started. The placebo group's score changed by −4.79 points on average, while the pridopidine groups changed by −3.37 (45 mg), −3.09 (67.5 mg), −4.13 (90 mg), and −2.74 (112.5 mg). Regarding adverse events (unwanted health occurrences reported during the trial), the reported data shows that 62 of 82 placebo participants, and between 63 and 71 of 81–82 participants in each pridopidine dose group, experienced at least one adverse event. For a pre-specified secondary measure of daily functioning (Total Functional Capacity, scored 0–13, where lower is worse) at 52 weeks, score changes ranged from −0.83 in the placebo group to −0.72, −0.65, and −0.59 in three of the pridopidine groups, while the 45 mg group showed a change of +0.04. Some additional post-hoc analyses (analyses planned after the trial completed) looked specifically at participants with earlier-stage Huntington's disease, but these involved only a subset of the data and their full details were not reported across all dose 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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• NCT03342053 · results posted 19 January 2021

  According to the results reported on ClinicalTrials.gov, this trial (NCT03342053) involved 46 people in total — 23 who received the study drug RO7234292 once a month, and 23 who received it once every two months. The drug was delivered directly into the fluid around the spine (a method called intrathecal administration). The trial was measuring, among other things, how often participants experienced adverse events (unwanted health events that occurred during the study), as well as levels of a specific protein in the spinal fluid, and changes in brain structure over 15 months. The reported data shows that, for adverse events — the primary thing the trial was tracking — 100% of participants in the monthly group and 95.7% in the every-two-months group had at least one adverse event recorded during the study. The reported data also shows changes in levels of a mutant protein (mHTT) in the spinal fluid, which the trial was measuring as a secondary outcome. Across various time points, both groups showed reductions in this protein compared to their starting levels, with the monthly group generally showing larger reductions. For example, at one measured time point the monthly group showed a reduction of around 50% and the every-two-months group around 33%, though the numbers varied across different time points. Regarding brain volume changes measured over 15 months, the reported data shows percentage changes in several brain regions for both groups, but what these numbers mean clinically was not described in the submitted results data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00652457 · results posted 5 January 2021

  According to the results reported on ClinicalTrials.gov, this trial involved 50 people in total — 23 in one group and 27 in another. The study used a "crossover" design, meaning one group received the medication memantine throughout the trial, while the other group started on a placebo (an inactive treatment) before switching to memantine. All 50 participants completed the study with no drop-outs recorded. The trial was measuring cognitive function — specifically memory — using a test called the Hopkins Verbal Learning Test – Revised (HVLT-R), as well as behavioural symptoms using a tool called the Neuropsychiatric Inventory (NPI). The reported data shows that no numerical results were submitted to ClinicalTrials.gov for either the primary outcome (the memory test) or the secondary outcome (the behavioural symptoms measure). For both measures, the actual scores or changes in scores were not reported in the structured results data available on ClinicalTrials.gov. Because no outcome numbers were provided in the submitted data, it is not possible to describe what the measurements showed for either 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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• NCT01897896 · results posted 16 April 2019

  According to the results reported on ClinicalTrials.gov, this trial (NCT01897896) looked at a medication called SD-809 ER in people with a movement disorder. There were two groups of participants: an 82-person "Rollover Cohort" (people who had been in an earlier related trial and continued onto this one) and a 37-person "Switch Cohort" (people who switched from a different form of the medication). In total, 119 people started the study. Of those, 56 people in the Rollover Cohort and 25 in the Switch Cohort completed it. The trial's main focus was on tracking and counting any unwanted medical events (called "treatment-emergent adverse events," or TEAEs) that participants experienced while taking the medication — not on measuring whether symptoms improved. The reported data shows that, looking at the entire treatment period, 77 out of 82 people in the Rollover Cohort and 35 out of 37 in the Switch Cohort experienced at least one TEAE of any kind. Of those, 21 (Rollover) and 11 (Switch) had a serious TEAE — meaning an event serious enough to involve hospitalisation, a life-threatening situation, or similar. Severe TEAEs (those that stopped participants from carrying out normal daily activities) were reported in 17 (Rollover) and 7 (Switch) people. TEAEs considered possibly related to the study drug were reported in 56 (Rollover) and 26 (Switch) participants. TEAEs that led a participant to withdraw from the study were reported in 13 (Rollover) and 3 (Switch) people. The reported data also shows small changes in routine blood test results (such as white blood cell counts) at week 158, though the clinical meaning of those numbers was not explained in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01806896 · results posted 14 December 2017

  According to the results reported on ClinicalTrials.gov, this trial involved 37 people in total — 20 were assigned to take PF-02545920 (20 mg twice a day) and 17 were assigned to take a placebo (a dummy pill with no active ingredient), also twice a day. The trial was primarily measuring safety-related observations, including any unwanted medical events that occurred during the study, unusual results on blood and urine tests, changes in heart readings (ECGs), changes in blood pressure and pulse, and changes in body weight. The reported data shows that when it came to unwanted medical events (called adverse events), 18 out of 19 people in the PF-02545920 group and 14 out of 17 people in the placebo group experienced at least one such event. One person in each group experienced a serious adverse event — meaning an event considered medically significant, such as hospitalisation. For laboratory test results flagged as potentially concerning, the reported data shows 8 participants in each group met that threshold. For heart trace (ECG) readings, small numbers of participants in both groups had findings noted — for example, 2 people in the PF-02545920 group and none in the placebo group had a particular type of heart interval reading flag raised. Regarding blood pressure and pulse readings of potential concern, the numbers reported were generally low across both groups, with most categories showing 0 to 2 participants affected. No participants in either group had a body weight change of 7% or more, and no participants met the blood count thresholds that would have required them to stop taking the study treatment. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00920699 · results posted 25 September 2017

  According to the results reported on ClinicalTrials.gov, this trial enrolled 91 people in total, split across three groups based on their daily dose of a supplement called CoQ10 (Coenzyme Q10): 29 people took 600 mg per day, 30 took 1,200 mg per day, and 32 took 2,400 mg per day. The trial was primarily measuring tolerability — that is, how many participants were able to stay on their originally assigned dose throughout the study without any changes to it. It also measured two things in the blood: levels of CoQ10 itself, and levels of a substance called 8OHdG, which the researchers were tracking as a marker of a certain type of cellular process. The reported data shows that, for the primary measure of tolerability, 90% of participants in the 600 mg group, 93.1% in the 1,200 mg group, and 81.3% in the 2,400 mg group completed the study on their original dose without modification. For the blood marker 8OHdG, the reported changes from the start of the study were a small increase of 0.15 ng/ml in the 600 mg group, a decrease of 1.56 ng/ml in the 1,200 mg group, and a small increase of 0.55 ng/ml in the 2,400 mg group. For CoQ10 blood levels, the reported changes were increases of 1.82 ng/ml, 1.92 ng/ml, and 2.33 ng/ml in the 600 mg, 1,200 mg, and 2,400 mg groups respectively. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT01795859 · results posted 11 August 2017

  According to the results reported on ClinicalTrials.gov, this trial (NCT01795859) involved 90 people in total — 45 who received a tablet called SD-809 and 45 who received a placebo (a dummy tablet with no active ingredient). Nearly all participants finished the trial: 44 in the SD-809 group and 43 in the placebo group. The trial was measuring changes in involuntary movements (called chorea) in people with Huntington's disease, using a scoring system called the Total Maximal Chorea (TMC) scale, which runs from 0 to 28, where a lower score means fewer involuntary movements. The reported data shows that, on the primary measure, the SD-809 group's TMC score decreased by an average of 4.42 points from their starting score, while the placebo group's score decreased by an average of 1.93 points. For the secondary measures, participants were also asked to rate their own overall change (called the Patient Global Impression of Change, or PGIC) — 23 out of 45 people in the SD-809 group rated themselves as "much" or "very much" improved, compared with 9 out of 45 in the placebo group. Clinicians made a similar assessment (called the CGIC) — 19 out of 45 in the SD-809 group were rated as much or very much improved, versus 6 out of 45 in the placebo group. On a physical functioning quality-of-life questionnaire (SF-36, scored 0–100 where higher means less disability), the SD-809 group's score changed by +0.74 points and the placebo group's by −3.61 points. On a balance test scored 0–56 (higher meaning better balance), the SD-809 group's score changed by +2.2 points and the placebo group's by +1.3 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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• NCT01590888 · results posted 18 July 2016

  According to the results reported on ClinicalTrials.gov, this trial (NCT01590888) looked at a drug called PBT2 in people with Huntington's disease. A total of 109 people took part — 36 received the higher dose of PBT2 (250 mg), 38 received the lower dose (100 mg), and 35 received a sugar pill (placebo). The trial's main goal was to measure how many participants in each group experienced at least one unwanted or unexpected health event (called an adverse event) while taking the treatment. It also measured changes in thinking skills, movement, everyday functioning, behaviour, and an overall impression of how participants were doing. The reported data shows that, for the primary measure — the number of people who experienced at least one adverse event — 32 out of 36 in the 250 mg group, 30 out of 38 in the 100 mg group, and 28 out of 35 in the sugar pill group reported at least one such event. For the thinking skills tests, scores are expressed as "z-scores" — a way of comparing individual results to an average, where a higher number means a better result relative to that average. The reported change from the start of the trial showed a small positive shift for the 250 mg group (around +0.06 to +0.23 depending on the test grouping), while the 100 mg and sugar pill groups showed small negative shifts. For movement, the 250 mg group showed a small decrease in their score of −0.7 (lower scores mean less impairment), the sugar pill group showed −1.3, and the 100 mg group showed +1.3. Changes in everyday functioning were similar across all three groups (around +1.1 to +1.3). Behaviour scores changed by −2.3 for the 250 mg group, +0.7 for the sugar pill group, and +3.0 for the 100 mg group (where lower scores indicate less disturbance). The overall investigator impression score — which weighs perceived benefit against side effects — was reported as 1.31 for the 250 mg group, 1.28 for the 100 mg group, and 1.18 for the sugar pill group; all values above 1 indicate the investigator judged some therapeutic effect was present. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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• NCT00608881 · results posted 30 March 2016

  According to the results reported on ClinicalTrials.gov, this trial looked at a high-dose form of a supplement called Coenzyme Q10 (2,400 mg per day) compared to a placebo (a dummy treatment with no active ingredient) in people with Huntington's disease. A total of 609 people took part — 303 in the Coenzyme Q10 group and 306 in the placebo group. The trial ran for 60 months (five years). The main thing being measured was a combined score that took into account both how long participants survived and how well they could manage everyday tasks and independent living — this was called a "joint rank" score. The reported data shows that on the main joint rank measure, the Coenzyme Q10 group received an average rank of 303.3 and the placebo group received an average rank of 306.7 — figures that were very close together. For the secondary measures, both groups showed declines over five years across all areas tested. The reported data shows that scores measuring ability to manage daily activities (Total Functional Capacity) fell by an average of 4.53 points in the Coenzyme Q10 group and 4.76 points in the placebo group (on a 0–13 scale). A daily task checklist (0–25 scale) dropped by 7.93 versus 8.02 points respectively. An independence scale (0–100) fell by 26.30 points in the Coenzyme Q10 group and 24.86 in the placebo group. A motor (movement) skills score (0–124, higher meaning more impairment) increased by 18.06 versus 19.18 points. Scores measuring behavioural symptoms increased by 1.39 versus 1.43 points across the two 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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• NCT01882062 · results posted 24 March 2016

  According to the results reported on ClinicalTrials.gov, this trial enrolled 10 participants, and all 10 completed the study. The trial was looking at the effect of a dietary supplement called triheptanoin, taken at a dose of 1 gram per kilogram of body weight per day for one month. The main thing being measured was how the brain uses energy — specifically, a ratio called Pi/PCr (the balance between two energy-related chemicals in the brain, inorganic phosphate and phosphocreatine). This ratio is used as a way of gauging how well the brain's energy-producing processes are working during activity. The reported data shows that the Pi/PCr ratio was measured at multiple time points across the study. The six reported values were 0.44, 0.43, 0.44, 0.42, 0.45, and 0.42. These numbers represent the balance between the two brain energy chemicals at different measurement occasions. It is worth noting that the data as submitted does not include a comparison group (for example, a placebo group), and no further breakdown — such as before-and-after comparisons or averages with variability — was reported in the structured results on ClinicalTrials.gov. Any figures not included in the submitted data 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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• NCT00277355 · results posted 19 April 2013

  According to the results reported on ClinicalTrials.gov, this trial looked at whether minocycline (an antibiotic taken at 100 mg twice a day) might slow the progression of Huntington's disease compared to a placebo (a dummy pill with no active ingredient). A total of 114 people took part — 87 in the minocycline group and 27 in the placebo group. Of those, 73 in the minocycline group and 22 in the placebo group completed the study. The main thing being measured was change in something called the Total Functional Capacity (TFC) scale, which is part of a standard Huntington's disease rating tool. The TFC scores a person's ability to manage everyday tasks like work, finances, household duties, and independent living, on a scale from 0 (greatest difficulty) to 13 (least difficulty). A higher change score over time would suggest greater decline. The reported data shows that over 18 months, the average decline in TFC score was 1.55 points in the minocycline group and 1.15 points in the placebo group, using a standard method to account for participants who did not complete the study. A secondary analysis using a different method to handle missing data reported a decline of 1.71 points in the minocycline group and 1.15 points in the placebo group. In both analyses, the minocycline group showed a slightly larger average decline in TFC score compared to the placebo group, though what this difference means clinically was not described in the submitted results data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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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.