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ClinicalTrials.gov public records Last synced Nov 15, 2026, 4:42 AM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 62 matching trials from the live ClinicalTrials.gov search.

Filters: Sponsor: PTC Therapeutics Clear all

Conditions: Duchenne Muscular Dystrophy
Interventions: Deflazacort; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 4 Years and older · Male only
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 4
States / cities: Los Angeles, California • Chicago, Illinois • Rochester, New York + 1 more

Conditions: Duchenne Muscular Dystrophy
Interventions: Ataluren; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: Male only
Enrollment: 265 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: Neurofibromatosis 2
Interventions: PTC299; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2012
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Duchenne Muscular Dystrophy
Interventions: PTC124; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 5 Years and older · Male only
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2007
U.S. locations: 3
States / cities: Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah

Conditions: Duchenne Muscular Dystrophy
Interventions: Ataluren; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019
U.S. locations: 1
States / cities: Los Angeles, California

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: AADC Deficiency
Interventions: Eladocagene Exuparvovec; Genetic
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 1 Year to 17 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2028
U.S. locations: 4
States / cities: Boston, Massachusetts • Durham, North Carolina • Cincinnati, Ohio + 1 more

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: Epilepsy
Interventions: ataluren, Placebo; Drug
Lead sponsor: NYU Langone Health; Other
Eligibility: 2 Years to 12 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 1
States / cities: New York, New York

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: 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: 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: 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: Cystic Fibrosis
Interventions: Ataluren; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 6 Years and older
Enrollment: 246 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 24
States / cities: Birmingham, Alabama • Long Beach, California • Los Angeles, California + 18 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: Leiomyosarcoma
Interventions: Unesbulin, Dacarbazine; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2024
U.S. locations: 4
States / cities: Jacksonville, Florida • Baltimore, Maryland • St Louis, Missouri + 1 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: 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

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: 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: 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

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: 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; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 7 Years to 15 Years · Male only
Enrollment: 219 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 21
States / cities: Los Angeles, California • Sacramento, California • Stanford, California + 18 more

Conditions: Kaposi's Sarcoma
Interventions: VEGF inhibitor PTC299, gene expression analysis, polymerase chain reaction, protein expression analysis, immunohistochemistry staining method, laboratory biomarker analysis, pharmacological study, biopsy; Drug · Genetic · Other + 1 more
Lead sponsor: AIDS Malignancy Consortium; Network
Eligibility: 18 Years to 120 Years
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 8
States / cities: La Jolla, California • Los Angeles, California • Honolulu, Hawaii + 4 more

Search by objective public record fields.

An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

PTC299 for Treatment of Neurofibromatosis Type 2

Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome

Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics

Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)

Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers

A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Extension Study of Ataluren (PTC124) in Cystic Fibrosis

Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma