Status:
AVAILABLE
Vatiquinone Expanded Access Protocol
Lead Sponsor:
Medical University of South Carolina
Collaborating Sponsors:
PTC Therapeutics
Conditions:
Mitochondrial Disease
Eligibility:
All Genders
1+ years
Brief Summary
The purpose of this study is to provide participants who successfully completed the study titled "An Open-label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitoch...
Detailed Description
This expanded access protocol serves to make Vatiquinone accessible to subjects with inherited mitochondrial disease who previously participated in a Vatiquinone clinical trial at the Medical Universi...
Eligibility Criteria
Inclusion
- Subjects with molecular genetic mitochondrial disease including Leigh syndrome, and Alpers syndrome.
- Completed participation in the study titled "An Open-label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease"
- In the opinion of the clinical investigator, the continuation to receive Vatiquinone will be of benefit to the participants.
- Enrollment into a clinical trial to receive Vatiquinone is not possible
Exclusion
- Current participation in any other interventional study
- Pregnant or currently breast feeding.
Key Trial Info
Start Date :
Trial Type :
EXPANDED_ACCESS
End Date :
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT07159139
Last Update
September 8 2025
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