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NCT05218655 · ClinicalTrials.gov registry record · Phase 3
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
A Phase 3 study, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 101
- Enrollment target
NCT05218655: Completed Phase 3 study, sponsored by PTC Therapeutics.
NCT05218655 is a Phase 3 study that has completed, run by PTC Therapeutics. The registered enrollment target is 101 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (87% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05218655, a Phase 3 study, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 101 participants
- Enrollment target
Study Summary
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical study or treatment plan. The study will continue until vatiquinone becomes commercially available or the program is terminated.
Primary Outcome
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included both serious adverse events (SAEs) and non-serious AEs. A TEAE was defined as an AE that had an onset date or date of worsening on or after the first dose of study drug and within 30 days of the date of the last dose of treatment. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE
Interventions
- DRUG Vatiquinone
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 101 participants |
| Start Date | 2022-06-22 |
| Est. Completion | 2025-04-15 |
| Phase | Phase 3 |
What the finished NCT05218655 record still lists
NCT05218655 is an interventional study that assigns participants to a tested intervention. The registered 101 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (87% lower).
The record links to 0 conditions, and to 1 intervention - of which Vatiquinone is the first listed.
NCT05218655 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT05218655 about?
NCT05218655 is a clinical study titled "A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease". The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical study or treatment plan. The study will continue until vatiquinone become...
What is the current status of trial NCT05218655?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 101 participants. The study started on 2022-06-22. Estimated completion is 2025-04-15.
What interventions are being tested in trial NCT05218655?
The interventions under investigation include: Vatiquinone (DRUG).
Who is sponsoring clinical trial NCT05218655?
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 NCT05218655's enrollment target sits among peer trials
101 1680th of 2000 higher than 320 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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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