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NCT04577352 · ClinicalTrials.gov registry record · Phase 2
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
A Phase 2 study, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 146
- Enrollment target
NCT04577352: Completed Phase 2 study, sponsored by PTC Therapeutics.
NCT04577352 is a Phase 2 study that has completed, run by PTC Therapeutics. The registered enrollment target is 146 participants, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (10% higher). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04577352, a Phase 2 study, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 146 participants
- Enrollment target
Study Summary
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale \[mFARS\]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
Primary Outcome
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
Interventions
- DRUG Placebo
- DRUG Vatiquinone
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 146 participants |
| Start Date | 2020-12-17 |
| Est. Completion | 2023-10-02 |
| Phase | Phase 2 |
What the finished NCT04577352 record still lists
NCT04577352 is an interventional study that assigns participants to a tested intervention. The registered 146 participants enrollment target is mid-sized for trials with a published cap, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (10% higher).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT04577352 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04577352 about?
NCT04577352 is a clinical study titled "A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia". The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale \[mFARS\]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
What is the current status of trial NCT04577352?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 146 participants. The study started on 2020-12-17. Estimated completion is 2023-10-02.
What interventions are being tested in trial NCT04577352?
The interventions under investigation include: Placebo (DRUG), Vatiquinone (DRUG).
Who is sponsoring clinical trial NCT04577352?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04577352's enrollment target sits among peer trials
146 496th of 2000 higher than 1,503 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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