At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 101 enrolled
Drug / intervention
Vatiquinonedrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease
In Brief
A Phase 3 clinical trial evaluating Vatiquinone for Inherited Mitochondrial Disease. Completed, enrolled 101 participants across 26 sites in 7 countries.
Detailed 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.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsInherited Mitochondrial Disease
CountriesFrance, Italy, Japan, Poland, Spain, United Kingdom, United States
Collaborators--
Timeline
Phase 3CompletedFinished
20222023202420252026
First PostedFeb 2022
Enrollment StartJun 2022
Primary CompletionApr 2025
TodayJul 2026
First PostedFeb 1, 2022
Enrollment StartJun 22, 2022
Primary CompletionApr 15, 2025
TodayJul 2, 2026
Enrollment to primary: 2.8 yearsPosted 4.4 years ago
Interventions
Vatiquinonedrug
Vatiquinone will be administered per dose and schedule specified in the arm description.