- Conditions
- Mitochondrial Disease, Neurology, Myopathy
- Interventions
- EPI-743, Placebo
- Drug
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2019
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–17
of 17
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- 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
- 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
- Parkinson's Disease
- Interventions
- EPI-743 400mg, EPI-743 200mg
- Drug
- Lead sponsor
- University of South Florida
- Other
- Eligibility
- 40 Years to 75 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Friedreich's Ataxia
- Interventions
- Placebo, EPI-743 400 mg, EPI-743 200 mg
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Tampa, Florida • Philadelphia, Pennsylvania
- 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
- 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
- Friedreich's Ataxia
- Interventions
- EPI-743
- Drug
- Lead sponsor
- University of South Florida
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Mitochondrial Diseases
- Interventions
- EPI-743
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 1 Year and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 16
- States / cities
- Los Angeles, California • Orange, California • Palo Alto, California + 12 more
- Conditions
- Leber's Hereditary Optic Neuropathy
- Interventions
- EPI-743
- Drug
- Lead sponsor
- Edison Pharmaceuticals Inc
- Industry
- Eligibility
- 8 Years to 65 Years
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- 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
- Conditions
- Noise-induced Hearing Loss
- Interventions
- EPI-743, Placebo
- Drug
- Lead sponsor
- Edison Pharmaceuticals Inc
- Industry
- Eligibility
- 18 Years to 30 Years
- Enrollment
- 77 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2016
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Friedreich's Ataxia
- Interventions
- Vatiquinone
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 7 Years to 21 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Tampa, Florida • Iowa City, Iowa + 1 more
- Conditions
- Tourette Syndrome
- Interventions
- EPI-743
- Drug
- Lead sponsor
- Yale University
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 1
- States / cities
- New Haven, Connecticut
- Conditions
- Friedreich Ataxia
- Interventions
- Vatiquinone, Placebo
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 7 Years and older
- Enrollment
- 146 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Gainesville, Florida • Tampa, Florida + 2 more
- Conditions
- Inherited Mitochondrial Disease
- Interventions
- Vatiquinone
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 101 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 14
- States / cities
- La Jolla, California • Stanford, California • New Haven, Connecticut + 10 more
- 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