- Conditions
- Friedreich Ataxia
- Interventions
- Vatiquinone
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Up to 6 Years
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–6
of 6
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Leigh Syndrome
- Interventions
- EPI-743
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 1 Year to 18 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2023
- U.S. locations
- 4
- States / cities
- Stanford University, California • Akron, Ohio • Houston, Texas + 1 more
- Conditions
- Leigh Syndrome
- Interventions
- Placebo, EPI-743 15 mg/kg, EPI-743 5 mg/kg
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 4
- States / cities
- Palo Alto, California • Akron, Ohio • Houston, Texas + 1 more
- Conditions
- Pearson Syndrome
- Interventions
- EPI-743
- Drug
- Lead sponsor
- Edison Pharmaceuticals Inc
- Industry
- Eligibility
- Up to 18 Years
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 4
- States / cities
- Orange, California • Cleveland, Ohio • Pittsburgh, Pennsylvania + 1 more
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome, Mitochondrial Encephalopathy (MELAS), Pontocerebellar Hypoplasia Type 6 (PCH6), Alpers Disease, Alpers Syndrome
- Interventions
- Vatiquinone, Placebo
- Drug · Other
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Up to 20 Years
- Enrollment
- 68 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 12
- States / cities
- San Diego, California • Stanford, California • New Haven, Connecticut + 7 more
- Conditions
- Friedreich Ataxia
- Interventions
- Vatiquinone
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 130 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Gainesville, Florida • Tampa, Florida + 2 more