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NCT04378075 · ClinicalTrials.gov registry record · Phase 2
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
A Phase 2 study, sponsored by PTC Therapeutics.
- Terminated
- Registry status
- Phase 2
- Development phase
- 68
- Enrollment target
NCT04378075: Clinical Trial Phase 2 study, sponsored by PTC Therapeutics.
NCT04378075 is a Phase 2 study that was terminated before completion, run by PTC Therapeutics. The registered enrollment target is 68 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (49% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04378075, a Phase 2 study, was terminated before completion, sponsored by PTC Therapeutics.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 68 participants
- Enrollment target
Study Summary
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants may enter a 48-week, long-term, extension phase during which they will receive open-label treatment with vatiquinone.
Primary Outcome
The 28-day motor seizure frequency in the double-blind period was calculated as the (number of motor seizures)/ (the number of valid days where motor seizure count information is present) \* 28 within the double-blind period. The baseline of the seizure frequency used the 28 days observations immediately prior to treatment start date for this calculation.
Interventions
- OTHER Placebo
- DRUG Vatiquinone
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 68 participants |
| Start Date | 2020-09-28 |
| Est. Completion | 2023-12-27 |
| Phase | Phase 2 |
Why NCT04378075 stopped before completion
NCT04378075 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 68 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (49% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT04378075 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04378075 about?
NCT04378075 is a clinical study titled "A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy". This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants m...
What is the current status of trial NCT04378075?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 68 participants. The study started on 2020-09-28. Estimated completion is 2023-12-27.
What interventions are being tested in trial NCT04378075?
The interventions under investigation include: Placebo (OTHER), Vatiquinone (DRUG).
Who is sponsoring clinical trial NCT04378075?
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 NCT04378075's enrollment target sits among peer trials
68 967th of 2000 higher than 1,027 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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