A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
- Conditions
- Inherited Mitochondrial Disease
- Interventions
- Registration Number
- NCT05218655
- Lead Sponsor
- PTC Therapeutics
- 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 (previously Edison) clinical study or treatment plan.
The study will continue until vatiquinone becomes commercially available or the program is terminated.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 102
- 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.
- Current participation in any other interventional study.
- Pregnancy or breast feeding.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Vatiquinone Vatiquinone Participants will receive vatiquinone oral solution (100 milligrams \[mg\]/milliliter \[mL\]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).
- Primary Outcome Measures
Name Time Method Number of Participants With Adverse Events (AEs) Baseline (Day 1) up to end of study (up to approximately 3 years)
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (26)
The Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
Children's Hospital of Philadelphia - CHOP
🇺🇸Philadelphia, Pennsylvania, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
Stanford University
🇺🇸Stanford, California, United States
Child Neurology Center of Northwest Florida
🇺🇸Gulf Breeze, Florida, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Ospedale Pediatrico Bambino Gesù
🇮🇹Roma, Italy
Hopital Necker-Enfants Malades
🇫🇷Paris, France
Seattle Children Hospital
🇺🇸Seattle, Washington, United States
PTC Clinical Site
🇯🇵Japanese City, Japan
University of California, San Diego Altman Clinical and Translational Research Institute
🇺🇸La Jolla, California, United States
Columbia University Medical Center - CUMC
🇺🇸New York, New York, United States
Chu Angers
🇫🇷Angers Cedex 9, France
CHU Montpellier - Hopital Saint-Eloi
🇫🇷Montpellier Cedex 5, France
Hôpital de Hautepierre - Hôpitaux Universitaires de Strasbourg
🇫🇷Strasbourg, France
Hospital Sant Joan de Déu
🇪🇸Barcelona, Spain
Great Ormond Street Hospital for Children NHS Foundation Trust
🇬🇧London, United Kingdom
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
Hospital Ruber Internacional
🇪🇸Madrid, Spain
UT Health The University of Texas
🇺🇸Houston, Texas, United States
Children's National
🇺🇸Washington, District of Columbia, United States
The Newcastle Upon Tyne Hospitals NHS Foundation Trust
🇬🇧Newcastle Upon Tyne, United Kingdom
Instytut Pomnik - Centrum Zdrowia Dziecka
🇵🇱Warszawa, Poland
Yale Medicine
🇺🇸New Haven, Connecticut, United States
Medical University of South Carolina - MUSC
🇺🇸Charleston, South Carolina, United States