Status:
COMPLETED
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
Lead Sponsor:
PTC Therapeutics
Conditions:
Inherited Mitochondrial Disease
Eligibility:
All Genders
Phase:
PHASE3
Brief 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 (pr...
Eligibility Criteria
Inclusion
- Participants with inherited mitochondrial disease including Leigh syndrome, Alpers syndrome, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonic epilepsy with ragged-red fibers (MERRF), pontocerebellar hypoplasia type 6 (PCH6), or other mitochondrial disease who participated in a previous vatiquinone clinical study or treatment plan.
- Women of childbearing potential must have a negative pregnancy test at screening/baseline and agree to abstinence or the use of at least 1 of the highly effective forms of contraception as specified in the protocol (with a failure rate of \<1% per year when used consistently and correctly). Highly effective contraception or abstinence must be continued for the duration of the study, and for up to 30 days after the last dose of study drug.
- Fertile men who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 30 days after the last dose of study drug.
Exclusion
- Current participation in any other interventional study.
- Pregnancy or breast feeding.
Key Trial Info
Start Date :
June 22 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 15 2025
Estimated Enrollment :
101 Patients enrolled
Trial Details
Trial ID
NCT05218655
Start Date
June 22 2022
End Date
April 15 2025
Last Update
December 22 2025
Active Locations (26)
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1
University of California, San Diego Altman Clinical and Translational Research Institute
La Jolla, California, United States, 92037
2
Stanford University
Stanford, California, United States, 94305
3
Yale Medicine
New Haven, Connecticut, United States, 06511
4
Children's National
Washington D.C., District of Columbia, United States, 20010