- 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
Search Results
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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 1–24
of 61
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
- Interventions
- Ataluren
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 173 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 15
- States / cities
- Sacramento, California • Aurora, Colorado • Gulf Breeze, Florida + 12 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Deflazacort
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 4 Years to 16 Years · Male only
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Chicago, Illinois • Rochester, New York + 1 more
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- EPI-589
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 21 Years to 70 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 3
- States / cities
- Los Angeles, California • San Francisco, California • Portland, Oregon
- Conditions
- Renal Impairment
- Interventions
- Deflazacort
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2015
- U.S. locations
- 2
- States / cities
- Miami, Florida • Orlando, Florida
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
- Interventions
- Ataluren, PLACEBO
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 360 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 22
- States / cities
- Phoenix, Arizona • Los Angeles, California • Oakland, California + 19 more
- Conditions
- Muscular Atrophy, Spinal
- Interventions
- Not listed
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- Not listed
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2014
- U.S. locations
- 2
- States / cities
- Kalamazoo, Michigan • Salt Lake City, Utah
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
- Interventions
- Ataluren, Placebo
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 7 Years to 16 Years · Male only
- Enrollment
- 230 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 21
- States / cities
- Los Angeles, California • Sacramento, California • Aurora, Colorado + 17 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Ataluren
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Male only
- Enrollment
- 270 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2026
- U.S. locations
- 29
- States / cities
- Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 25 more
- Conditions
- Hemophilia A, Hemophilia B
- Interventions
- Ataluren
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 7
- States / cities
- Peoria, Illinois • Indianapolis, Indiana • Worcester, Massachusetts + 4 more
- Conditions
- Phenylketonuria
- Interventions
- PTC923
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 247 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 7
- States / cities
- Aurora, Colorado • Gainesville, Florida • Indianapolis, Indiana + 4 more
- Conditions
- Cystic Fibrosis
- Interventions
- Ataluren
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 191 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 15
- States / cities
- Birmingham, Alabama • Long Beach, California • Palo Alto, California + 12 more
- Conditions
- Phenylketonuria
- Interventions
- PTC923, Placebo
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 157 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 12
- States / cities
- Stanford, California • Aurora, Colorado • Gainesville, Florida + 9 more
- Conditions
- Healthy Volunteers
- Interventions
- Deflazacort
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015
- U.S. locations
- 1
- States / cities
- San Antonio, Texas
- Conditions
- Phenylketonuria
- Interventions
- Sepiapterin
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Up to 9 Years
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2031
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- Conditions
- Leiomyosarcoma
- Interventions
- Unesbulin, Dacarbazine, Placebo
- Drug · Other
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 359 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 23
- States / cities
- Duarte, California • Los Angeles, California • Santa Monica, California + 18 more
- Conditions
- High Grade Glioma, Diffuse Intrinsic Pontine Glioma
- Interventions
- PTC596, Radiotherapy
- Drug · Radiation
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 12 Months to 21 Years
- Enrollment
- 64 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2029
- U.S. locations
- 10
- States / cities
- Aurora, Colorado • Washington D.C., District of Columbia • Chicago, Illinois + 7 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Ataluren
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Male only
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 3
- States / cities
- Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah
- Conditions
- Cystic Fibrosis
- Interventions
- PTC124
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2006
- U.S. locations
- 5
- States / cities
- Birmingham, Alabama • Palo Alto, California • Denver, Colorado + 2 more
- Conditions
- Healthy Volunteers
- Interventions
- Deflazacort and rifampin, Deflazacort and Clarithromycin
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 58 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014
- U.S. locations
- 1
- States / cities
- Tempe, Arizona
- Conditions
- Cystic Fibrosis
- Interventions
- Ataluren
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Long Beach, California • Aurora, Colorado + 4 more
- Conditions
- Limb-Girdle Muscular Dystrophy
- Interventions
- Deflazacort
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 8
- States / cities
- Atlanta, Georgia • Iowa City, Iowa • Kansas City, Kansas + 5 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
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
- Interventions
- Ataluren, Chronic Corticosteroid Therapy
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 7 Years and older · Male only
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010
- U.S. locations
- 5
- States / cities
- Davis, California • Boston, Massachusetts • Minneapolis, Minnesota + 2 more