Trial · NCT04577352
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT04577352, Phase 2, Phase 3, Completed, PTC Therapeutics
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia. Trials, record NCT04577352. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04577352
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
- NCT number
- NCT04577352
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 146 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Friedreich Ataxia
- Interventions
- Vatiquinone, Placebo
- First posted
- 2020-10-06
- Last updated
- 2026-04-13
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in the mFARS Score at Week 72 - Modified Intent-to-treat (mITT) Analysis Set | Baseline, Week 72 | mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. Least square (LS) mean and standard error (SE) was calculated using mixed-model repeated measures (MMRM). |
| Primary | Change From Baseline in the mFARS Score at Week 72 - Intent-to-treat (ITT) Analysis Set | Baseline, Week 72 | mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. |
| Secondary | Change From Baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) Score at Week 72 - mITT Analysis Set | Baseline, Week 72 | The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 4=severe disability/inability to carry out activity independently for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. |
| Secondary | Change From Baseline in FARS-ADL Score at Week 72 - ITT Analysis Set | Baseline, Week 72 | The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 4=severe disability/inability to carry out activity independently for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. |
| Secondary | Change From Baseline in 1-Minute Walk Test (1MWT) at Week 72 - mITT Analysis Set | Baseline, Week 72 | The 1MWT is a timed performance test used to measure functional ability, walking endurance, balance, and muscle performance by measuring maximal walking speed in 1 minute. Participants were instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed was measured upon completion of the walk and distance was recorded. The mean change from baseline in the distance the participant walked is reported. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. |
| Secondary | Change From Baseline in 1MWT at Week 72 - ITT Analysis Set | Baseline, Week 72 | The 1MWT is a timed performance test used to measure functional ability, walking endurance, balance, and muscle performance by measuring maximal walking speed in 1 minute. Participants were instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed was measured upon completion of the walk and distance was recorded. The mean change from baseline in the distance the participant walked is reported. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. |
| Secondary | Number of Falls Per 28 Days Over Every 24-Week Period - mITT Analysis Set | Week 1-24, Week 25-48, and Week 49-72 | Each participant was required to maintain a fall log, which included the date and time of each fall. Falls as defined by World Health Organization as "inadvertently coming to rest on the ground, floor or other lower level, excluding intentional change in position to rest in furniture, wall or other objects," were reported. Number of falls per 28 days during a time interval was calculated as the number of falls during the period divided by the number of days during the interval, and multiplied by 28. The falls that occurred on or after the first Loss of Ambulation visit were excluded from the analysis. |
| Secondary | Number of Falls Per 28 Days Over Every 24-Week Period - ITT Analysis Set | Week 1-24, Week 25-48, and Week 49-72 | Each participant was required to maintain a fall log, which included the date and time of each fall. Falls as defined by World Health Organization as "inadvertently coming to rest on the ground, floor or other lower level, excluding intentional change in position to rest in furniture, wall or other objects," were reported. Number of falls per 28 days during a time interval was calculated as the number of falls during the period divided by the number of days during the interval, and multiplied by 28. The falls that occurred on or after the first Loss of Ambulation visit were excluded from the analysis. |
| Other | Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - mITT Analysis Set | Baseline, Week 72 | mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. The Upright Stability subscale of the mFARS includes following items: 1. Sitting posture (score: 0 to 4); 2a. Stance with feet apart and eyes open (score: 0 to 4); 2b. Stance with feet apart and eyes closed (score: 0 to 4); 3a. Stance with feet together and eyes open (score: 0 to 4); 3b. Stance with feet together and eyes closed (score: 0 to 4); 4. Tandem stance (score: 0 to 4); 5. Stance on dominant foot (score: 0 to 4); 6. Tandem walk (score: 0 to 3); 7. Gait (score: 0 to 5). The upright stability subscale total score ranges from 0 (normal) to 36 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. |
| Other | Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - ITT Analysis Set | Baseline, Week 72 | mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. The Upright Stability subscale of the mFARS includes following items: 1. Sitting posture (score: 0 to 4); 2a. Stance with feet apart and eyes open (score: 0 to 4); 2b. Stance with feet apart and eyes closed (score: 0 to 4); 3a. Stance with feet together and eyes open (score: 0 to 4); 3b. Stance with feet together and eyes closed (score: 0 to 4); 4. Tandem stance (score: 0 to 4); 5. Stance on dominant foot (score: 0 to 4); 6. Tandem walk (score: 0 to 3); 7. Gait (score: 0 to 5). The upright stability subscale total score ranges from 0 (normal) to 36 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04577352
GET https://conductscience.org/api/trials/v1/NCT04577352/registered