- 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
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 1–17
of 17
matching trials from the live ClinicalTrials.gov search.
- 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
- 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
- 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
- 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
- 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
- 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
- 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'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
- 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
- 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'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
- 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
- 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
- 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
- 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