Reported trial results for Friedreich Ataxia
Every Friedreich Ataxia trial Voxsanity tracks that has reported results to a public registry, newest first. These trials have finished, so they are not recruiting. Not medical advice.
18 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
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NCT04577352 · results posted 8 April 2026
According to the results reported on ClinicalTrials.gov, this trial (NCT04577352) enrolled 146 people with Friedreich's ataxia — 73 in the vatiquinone group and 73 in the placebo group. The main 72-week blinded phase was completed by 63 people in the vatiquinone group and 65 in the placebo group. The trial was primarily measuring changes in a neurological test called the mFARS score, which rates things like balance, limb coordination, and speech on a scale of 0 (normal) to 93 (greatest impairment) — so a smaller increase over time means less worsening. There was also an additional 24-week open-label phase where all participants received vatiquinone. The reported data shows that over 72 weeks, scores in both groups increased from their starting points (meaning some worsening was recorded in both groups). In the primary analysis, the vatiquinone group's mFARS score increased by approximately 1.2 points on average, compared to approximately 2.8 points in the placebo group. A second way of counting all enrolled participants gave similar figures: roughly 0.9 points for vatiquinone versus 2.6 points for placebo. For the secondary measure of daily living activities (things like walking, dressing, and speech, scored 0–36), the reported average increases were approximately 0.8 points for vatiquinone and 1.7 points for placebo. In a one-minute walking test, the reported data shows both groups walked a shorter distance compared to their starting point: the vatiquinone group declined by about 4.3 metres on average, while the placebo group declined by about 9.3 metres 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.
Read the full trial page · View reported results on ClinicalTrials.gov ↗
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NCT04192136 · results posted 11 March 2026
According to the results reported on ClinicalTrials.gov, this trial (NCT04192136) enrolled 75 people in total across five groups. Four groups — comprising 66 participants — were randomly assigned to one of four combinations: a supplement called Nicotinamide Riboside (NR) alone, a placebo (inactive treatment) alone, exercise plus NR, or exercise plus placebo. A fifth group of 9 people were not randomised and did not complete the study. All 66 randomised participants finished the trial. The trial was measuring two main things: how well participants' hearts and lungs could use oxygen during exercise (called "peak VO2," measured in litres per minute), and how well their bodies responded to insulin (a hormone that controls blood sugar), using a score called the Whole Body Insulin Sensitivity Index. The reported data shows the following changes in peak oxygen uptake from the start to the end of the trial: the NR-only group increased by 0.06 L/min on average, the placebo-only group decreased by 0.06 L/min, the exercise-plus-NR group increased by 0.10 L/min, and the exercise-plus-placebo group increased by 0.03 L/min. For the insulin sensitivity score (a unitless number where a higher value means the body is responding better to insulin), the reported data shows changes of −0.23 for the NR-only group, −0.11 for the placebo-only group, −0.31 for the exercise-plus-NR group, and −0.51 for the exercise-plus-placebo group — meaning all four groups showed a small decrease in this score on average. No further statistical detail (such as whether these differences were considered meaningful by the researchers) was reported in the data provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03418740 · results posted 16 January 2025
According to the results reported on ClinicalTrials.gov, this trial enrolled 108 children with Friedreich's Ataxia (FA), a rare inherited condition that affects the nervous system and movement. Of those, 89 were included in the main analysis and 85 completed the study. The trial was not testing a treatment — rather, it was an observational study tracking how several measures of movement, balance, and daily functioning changed over three years in this group of children. Measurements were taken at the start of the study and then at one, two, and three years. The reported data shows that the primary measure — the modified Friedreich's Ataxia Rating Scale (mFARS), a scoring tool where higher numbers indicate greater difficulty with movement — increased by an average of 2.9 points after one year, 4.9 points after two years, and 7.7 points after three years. For the secondary measures, the reported data shows changes in a timed walking test (25 feet), a finger-dexterity peg test, and a timed stand-and-walk test were each small in numerical terms across the three years (for example, the stand-and-walk test changed by about 4.9 seconds by year three). A balance scale (where lower scores reflect more difficulty) showed an average decrease of 6.5 points at one year, 10.4 at two years, and 15 points at three years. A self-reported daily activities scale showed an average increase of 0.1 points at one year, rising to 3 points at three years, where higher scores indicate more difficulty. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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NCT04817111 · results posted 17 July 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT04817111) enrolled 7 adults who all received an experimental supplement called MIB-626. All 7 participants completed the study. The trial was a single-group, open-label study — meaning everyone received the same treatment and both participants and researchers knew what was being given. The main thing researchers were looking at was how many participants experienced unwanted side effects (called "adverse events") while taking MIB-626, graded using a standard medical rating scale. They also took a range of measurements before and after treatment, including specialised heart and muscle scans, grip strength, and levels of a substance in the blood called NAD+ (a molecule involved in how cells produce energy). The reported data shows that 4 out of the 7 participants had at least one treatment-related adverse event of Grade 1 or higher (Grade 1 being the mildest level on the rating scale). For the secondary measurements, the reported average change in a heart energy marker called the PCr/ATP ratio (a measure of how the heart uses energy, captured by a specialised scan) was −0.445, meaning it was lower after treatment on average. Average grip strength changed by −0.65 kg (also a small decrease). The average level of NAD+ in whole blood was reported as 49.9 micromoles per litre, though the data as submitted does not separately report the before-and-after values, only this single figure. The reported data shows no measurements were submitted for the muscle scan outcome (post-exercise CrCEST MRI), so those results were not reported. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT02255435 · results posted 29 November 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT02255435) involved people with Friedreich's ataxia, a rare condition affecting movement and coordination. The trial was run in two parts. Part 1 enrolled 69 participants across seven dose groups of a medicine called omaveloxolone (ranging from 2.5 mg up to 300 mg) plus a placebo group of 17 people, to explore different doses over 12 weeks. Part 2 enrolled 103 participants — 52 on placebo and 51 on a 150 mg dose of omaveloxolone — over 48 weeks. Part 1 looked mainly at how hard participants could work on a stationary exercise bike, while Part 2 focused on changes in a neurological function score called the mFARS, where a lower score means better function (the scale runs from 0 to 99). The reported data shows the following numbers. In Part 1, the change in exercise capacity (measured in watts per kilogram of body weight) after 12 weeks ranged from a small increase of 0.14 W/kg in the lowest dose group down to a small decrease of −0.09 W/kg in the 20 mg group; the placebo group showed a change of 0.04 W/kg. In Part 2, after 48 weeks the mFARS score in the placebo group increased by an average of 0.85 points (meaning slightly worse function on average), while the omaveloxolone 150 mg group showed an average change of −1.55 points (meaning a small improvement in score on average). For the secondary measure in Part 1 — the mFARS score at 12 weeks — all groups including placebo showed small reductions in score, ranging from −0.88 to −3.75 across the omaveloxolone doses, compared with −1.43 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.
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04102501 · results posted 27 May 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT04102501) involved 65 participants in total — 33 received a treatment called RT001 and 32 received a placebo (a dummy treatment with no active ingredient). The trial ran for 11 months and was measuring two things: how much oxygen the body can use at maximum effort during a supervised exercise test (known as a cardiopulmonary exercise test), and how far participants could walk in one minute. The reported data shows that for the main measure — the change in maximum oxygen use from the start of the trial to 11 months — the RT001 group showed a change of +0.05 ml of oxygen per kilogram of body weight per minute, while the placebo group showed a change of +0.08 ml of oxygen per kilogram per minute. For the secondary measure — the change in one-minute walking distance — the RT001 group showed a change of −193.83 inches and the placebo group showed a change of −220.42 inches. Both groups therefore showed a decrease in walking distance over the study period. It is worth noting that 8 people in the RT001 group and 12 people in the placebo group did not complete the trial, and no information about the reasons was reported in the data provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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NCT02660112 · results posted 5 December 2019
According to the results reported on ClinicalTrials.gov, this trial (NCT02660112) involved 10 participants, all of whom completed the study with no drop-outs. The trial was looking at a compound called (+)-Epicatechin and measuring two main things in people with Friedreich's Ataxia: changes in a disability rating scale called the FARS (Friedreich Ataxia Rating Scale), and changes in heart muscle thickness as seen on a cardiac MRI scan. The FARS score combines several tests — including walking speed, hand coordination, speech speed, and vision — into a single number ranging from 0 to 159, where a higher number means a greater level of disability. The reported data shows that the average FARS composite score recorded was 55.0 (out of a possible 159). For the heart measurement, the reported figure for left ventricular mass indexed to body surface area — a way of measuring heart muscle size relative to body size — was 55.9 mL/m². It is worth noting that the data as submitted appears to report the scores recorded during the study, but a clear "change from baseline" figure (that is, how much these numbers shifted from the start of the trial to the end) was not separately reported in the structured results data available on ClinicalTrials.gov. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03120013 · results posted 28 June 2019
According to the results reported on ClinicalTrials.gov, this trial involved 21 people in total (10 in one group, 11 in the other), all of whom had ataxia — a condition affecting balance and coordination. It was a "crossover" trial, meaning everyone received both the real treatment and a sham (dummy) version at different times, with a six-month rest period in between. The treatment being tested was tDCS (transcranial direct current stimulation), a technique that passes a very mild electrical current through the scalp. The trial measured changes in participants' ataxia symptoms using several rating tools and physical tasks. The reported data shows results from two main ataxia rating scales. On the ICARS scale (which runs from 0, meaning no ataxia, to 100, meaning the most severe ataxia), scores in the real tDCS group started at around 53 and were recorded at 43 at one time point, compared to the sham group whose scores stayed around 52–53 across the same time points. The secondary outcome data showed the real tDCS group's ICARS scores ranging from about 41 to 44 across multiple follow-up time points, while the sham group remained close to 52. On the SARA scale (0 to 40), the real tDCS group recorded a score of around 15.8 compared to 19.7 for the sham group at one key time point, with similar patterns seen across follow-up measurements. For the physical tasks — a peg test measuring hand coordination (in seconds) and an 8-metre walking test — the reported data shows the real tDCS group recorded lower (faster) times at most time points compared to the sham group, though the figures were not reported for every time point in the primary outcomes section. 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 ↗
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NCT02593773 · results posted 18 May 2018
According to the results reported on ClinicalTrials.gov, this trial (NCT02593773) enrolled 86 people, all of whom received the study drug Interferon γ-1b. The trial was an extension study, meaning participants had previously taken part in an earlier related trial. Of the 86 who started, 51 completed the study and 35 did not finish. The trial was measuring a range of things, including any unwanted medical events that occurred during treatment, whether the body produced antibodies (proteins) against the drug, and changes in scores on several rating scales that assess movement, coordination, and daily living activities in people with Friedreich's Ataxia — a condition that affects balance, coordination, and muscle control. The reported data shows that when it came to unwanted medical events, 78 out of 86 participants experienced at least one treatment-emergent adverse event (that is, any new or worsening health issue that appeared after starting the drug). Serious adverse events — defined as events involving hospitalisation, life-threatening situations, lasting disability, or death — were reported for 61 participants. Four participants stopped taking the drug because of an adverse event, and no deaths were reported as a serious adverse event in that category. Regarding antibodies against the drug, the data shows that at various points during the study, nearly all participants tested negative for anti-drug antibodies, with only 1 participant recording a positive result at the starting point. For the key rating scale scores measuring movement, coordination, and daily living abilities — which were listed as both primary and secondary outcomes — the reported data shows no numerical results were submitted to ClinicalTrials.gov 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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NCT02797080 · results posted 1 May 2018
According to the results reported on ClinicalTrials.gov, this trial (NCT02797080) involved 38 participants who all received a treatment called Interferon γ-1b. Of those 38 people, 36 completed the study and 2 did not finish. The trial was measuring how participants responded to the treatment in terms of unwanted medical events — specifically, it tracked what are called "treatment-emergent adverse events" (unexpected or unwanted health changes that occurred after starting the medication), "serious adverse events" (more severe health events such as hospitalisation or life-threatening situations), and whether anyone had to stop taking the treatment because of such an event. The reported data shows that out of the 38 participants, 21 experienced at least one treatment-emergent adverse event — that is, some kind of unwanted health change that occurred during the study period. Seven participants experienced a serious adverse event, meaning a more significant health concern such as requiring hospitalisation. The reported data shows that zero participants stopped taking the treatment due to an adverse event. The remaining measurement values in the data were also reported as zero, though the specific categories those zeros correspond to were not fully labelled in the data provided, so a detailed breakdown cannot be given here. It is worth noting that this trial only had one group (everyone received the same treatment), so there was no comparison group reported in this 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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NCT00993967 · results posted 5 March 2018
According to the results reported on ClinicalTrials.gov, this trial enrolled 200 people, all of whom received the study drug, idebenone. The trial was measuring two main things: first, how often and what kinds of unwanted effects (called adverse events) occurred; and second, whether there were any changes in a standard rating scale used to assess a type of movement disorder called ataxia — the International Cooperative Ataxia Rating Scale, or ICARS. The ICARS looks at things like balance, walking, coordination of the arms and legs, speech, and eye movements, and gives a total score from 0 (least affected) to 100 (most severely affected). Of the 200 people who started, 139 completed the trial and 61 did not. The reported data shows that, out of the 188 participants counted in the safety analysis, 109 experienced at least one adverse event. Of those, 54 experienced what were classified as serious adverse events, 8 experienced adverse events that led to stopping the study medication, 26 experienced adverse events considered possibly related to the study drug, and 1 participant passed away (though the data does not specify the cause or its relationship to the study drug). Regarding the ICARS movement scale, the reported data shows two measurements of 1.41 and 2.88 units — however, the data as submitted does not clearly label which figure represents which time point or comparison, so it is not possible to describe these numbers in fuller context without risking misrepresentation. It is also worth noting that several other planned safety measurements — including physical examinations, heart tracings (ECGs), and blood test results — were listed as planned outcomes in the trial, but no numerical results were reported for those measures 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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NCT01716221 · results posted 19 September 2016
According to the results reported on ClinicalTrials.gov, this trial enrolled just one participant, who completed the full study. The trial was looking at the effects of two medicines — bupropion and citalopram — on a condition called Friedreich's ataxia, a disease that affects movement and coordination. The participant went through several different treatment combinations over approximately 20 weeks, and their symptoms were tracked using two established rating scales: the International Cooperative Ataxia Rating Scale (ICARS, scored 0–100, where higher means more severe symptoms) and the Friedreich Ataxia Rating Scale (FARS, scored 0–159, where higher means more severe symptoms). The reported data shows the following ICARS scores across the different treatment phases: at baseline (before any treatment change) the score was 42; during bupropion combined with citalopram it was 39; during bupropion with a dummy pill (placebo) it was 30; during a dummy pill with citalopram it was 47; and during two dummy pills it was 46. For the FARS scale, the reported scores were: 43 at baseline, 41 at week 5 (citalopram only), 47 at week 10 (no active treatment), 42 at week 15 (bupropion only), and 45 at week 20 (bupropion plus citalopram). A depression questionnaire (Hamilton Depression Rating Scale, scored 0–66) was also completed, with scores ranging from 2 to 10 across the different phases; a score of 0–7 is considered normal on that scale. Because this trial involved only a single participant, the reported data shows the experience of that one person alone across different time points and treatment combinations. No data was reported that would allow any broader conclusions to be drawn from these numbers. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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NCT00905268 · results posted 27 June 2016
According to the results reported on ClinicalTrials.gov, this trial (NCT00905268) involved 232 people with Friedreich's ataxia — a condition affecting coordination and movement. Participants were divided into four groups: three groups received different doses of a medicine called idebenone (57, 57, and 59 people respectively), and one group of 59 people received a placebo (a dummy treatment with no active ingredient). The trial ran for 52 weeks and was primarily measuring changes in a coordination rating scale called ICARS, which runs from 0 to 100, where higher scores mean greater difficulty with movement and coordination. The reported data shows that on the primary measure — the ICARS scale — all four groups showed a small increase in score from the start of the trial to week 52. The three idebenone groups showed average increases of 1.6, 1.7, and 1.2 points respectively, while the placebo group showed an average increase of 1.1 points. On a related scale called FARS (which measures disability on a scale of 0 to 159), the reported changes were similarly small increases across all groups: 0.9, 1.2, and 1.4 points for the idebenone groups, and 0.9 points for the placebo group. For a secondary measure looking at the proportion of participants whose ICARS score improved by 2.5 points or more, the reported data shows 18.2%, 23.6%, and 23.7% of participants in the three idebenone groups met this threshold, compared with 31% in the placebo group. For heart-related measures in the subset of participants with cardiac involvement, the proportion showing improvement in a heart function measure ranged from 30.3% to 51.4% across idebenone groups, compared with 44.1% in the placebo group. Changes in exercise capacity (measured in Watts) varied across groups, and some figures were not clearly reported in a way that allows further plain description without risk of misrepresentation. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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NCT01303406 · results posted 9 March 2016
According to the results reported on ClinicalTrials.gov, this trial involved 29 people in total — 13 received a placebo (a dummy treatment with no active ingredient) and 16 received idebenone, a medicine being investigated for Friedreich's ataxia (FRDA), a rare inherited condition affecting movement and coordination. All 13 people in the placebo group and 15 of the 16 in the idebenone group completed the trial (one person in the idebenone group did not finish). The trial was primarily measuring whether participants could correctly guess which treatment they had received — placebo or idebenone — which can be a way of checking how "blinded" a trial is (meaning whether participants could tell the treatments apart). The reported data shows that, when asked whether they thought they had received idebenone, 6 out of 13 people in the placebo group said yes, and 8 out of 16 people in the idebenone group also said yes. A secondary measure looked at how many participants in each group left the trial early because their FRDA symptoms came back or got worse. The reported data shows that no participants in either group — 0% in the placebo group and 0% in the idebenone group — withdrew for this reason. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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NCT01965327 · results posted 30 April 2015
According to the results reported on ClinicalTrials.gov, this trial (NCT01965327) enrolled 12 people with Friedreich's ataxia, a rare inherited condition affecting the nervous system. All 12 participants received a treatment called Interferon Gamma-1b (also known as ACTIMMUNE). Ten participants completed the trial, while two did not finish. The trial was primarily measuring whether the treatment changed levels of a protein called frataxin in the blood — frataxin is produced at lower-than-normal levels in people with Friedreich's ataxia. A secondary goal was to track any change in participants' scores on the Friedreich Ataxia Rating Scale (FARS), a standardised tool used to assess things like balance, walking, coordination, speech, and daily activities, where higher scores indicate greater difficulty. The reported data shows that, on average, frataxin levels in the blood at the end of the 12-week treatment period were approximately 1.5% lower than they were at the start of the trial. For the secondary measure, the reported data shows an average change of about minus 4.98 points on the FARS scale (which runs from 0 to 159), meaning participants' scores were slightly lower at the end of the study compared to the beginning. Because a lower FARS score indicates less measured disability, this reflects the direction of change in the numbers reported — however, the data as submitted does not include further detail such as ranges or comparison groups that would provide broader context for 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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NCT00824512 · results posted 21 May 2013
According to the results reported on ClinicalTrials.gov, this trial enrolled 22 people in total — 11 in each group. One group took a 120 mg dose of a ginkgo biloba extract called EGb 761®, and the other group took a placebo (a dummy treatment). All 22 participants completed the trial. The study was measuring how well muscles recover after exercise, looking specifically at how quickly muscles restore their energy, how much blood flows to the muscle after exercise, how fast oxygen returns to the muscle, and the size of the muscle — all measured using specialised imaging and scanning techniques. The reported data shows the following numbers for the main measure — the rate at which muscles rebuild their energy supply after exercise (measured in units called "pH per second"): the EGb 761® group recorded 0.024 at the start and 0.022 at the end, while the placebo group recorded 0.029 at both time points. For the secondary measures, the peak blood flow to the muscle after exercise was reported as 54.30 (EGb 761®) versus 51.80 (placebo) at the start, and 58.80 versus 46.60 at the end. The time it took for blood flow to reach its peak was reported as 38.30 seconds (EGb 761®) versus 58.55 seconds (placebo) at the start, and 39.80 versus 76.55 seconds at the end. The rate at which oxygen returned to the muscle was 0.087 (EGb 761®) versus 0.054 (placebo) at the start, and 0.058 versus 0.062 at the end. Muscle size and total blood flow over nine minutes were also measured, with the reported figures showing only small differences between the two groups across both time 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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NCT00537680 · results posted 29 December 2011
According to the results reported on ClinicalTrials.gov, this trial looked at two different doses of a medicine called idebenone — a "mid dose" and a "high dose" — compared to a dummy treatment (placebo) in people with Friedreich's ataxia, a condition affecting movement and coordination. A total of 70 people started the trial (22 in the mid-dose group, 24 in the high-dose group, and 24 in the placebo group), and 68 completed it. The trial ran for 24 weeks and used several rating scales to measure changes in movement, coordination, daily activities, and heart structure. The reported data shows that the main thing being measured was a movement and coordination score called ICARS (scored from 0 to 100, where a higher number means greater difficulty). After 24 weeks, the mid-dose group's average score changed by minus 2.5 points, the high-dose group by minus 2.4 points, and the placebo group by minus 1.3 points — meaning all three groups showed some reduction in their scores from where they started. A second movement scale called FARS (scored 0 to 159) showed average changes of minus 1.6 points (mid dose), minus 1.2 points (high dose), and plus 0.6 points (placebo). For daily living activities (a sub-score of FARS, ranging 0 to 36), the reported changes were plus 0.2 (mid dose), plus 0.6 (high dose), and plus 1.0 (placebo) — meaning scores in all groups moved slightly in the direction of greater difficulty. Heart measurements (the thickness of the left ventricle wall) also showed small numerical differences between groups, but the clinical meaning of these figures was not reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT00697073 · results posted 4 August 2011
According to the results reported on ClinicalTrials.gov, this trial involved 68 people with Friedreich's ataxia who received a high dose of a medicine called idebenone. Of those 68 participants, 59 completed the study and 9 did not finish. The trial was measuring changes in two rating scales commonly used to assess Friedreich's ataxia — the ICARS (International Cooperative Ataxia Rating Scale, scored from 0 to 100, where a lower score means less disability) and the FARS (Friedreich's Ataxia Rating Scale, scored from 0 to 159, where a lower score also means less disability). The reported data shows that, on average, participants' ICARS scores changed by 1.1 units over the course of the study. Because a rise in this score indicates more disability, this means scores on average moved slightly in the direction of greater disability. Similarly, the reported data shows FARS scores changed by an average of 2.3 units, also in the direction of greater disability on average. It is important to note that these figures represent averages across the group, and individual results would have varied. The trial also tracked unwanted events (sometimes called "adverse events") experienced by participants. The reported data shows a total of 117 events were recorded across different categories, with separate figures of 30 mild, 32 moderate, and 4 severe events noted — though the full breakdown of how these figures relate to one another was not entirely clear from 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.
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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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.