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Showing 1–24 of 25 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: Ataluren Clear all
Completed Phase 3 Interventional Results available

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

NCT01826487
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 3, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Terminated Phase 2 Interventional Results available

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

NCT01009294
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 28, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 3 Interventional Results available

Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)

NCT02139306
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ataluren (PTC124®), Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
279 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
30
States / cities
Birmingham, Alabama • Mobile, Alabama • Long Beach, California + 23 more
Source: ClinicalTrials.gov public record
Updated May 13, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 4 Interventional Results available

Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations

NCT03256799
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor/Ataluren
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
19 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Jul 18, 2019 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 3 Interventional Results available

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Sep 3, 2026, 7:27 AM EDT
Terminated Phase 3 Interventional Results available

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

NCT02090959
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 10, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 2 Interventional Results available

Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

NCT00592553
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Ataluren, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
174 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
15
States / cities
Sacramento, California • Aurora, Colorado • Pensacola, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 3 Interventional

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

NCT01247207
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 8, 2026 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT04336826
View directory record Official record
Conditions
Nonsene Mutation Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
6 Months to 2 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Mar 31, 2024 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 2 Interventional Results available

Study of Ataluren in Participants With Nonsense Mutation Aniridia

NCT02647359
View directory record Official record
Conditions
Aniridia
Interventions
Ataluren, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
2 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
2
States / cities
Portland, Oregon • Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated May 26, 2022 · Synced Sep 3, 2026, 7:27 AM EDT
Terminated Phase 3 Interventional Results available

Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

NCT02456103
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 26, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Terminated Phase 2 Interventional Results available

Study of Ataluren (PTC124) in Hemophilia A and B

NCT00947193
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 15, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 2 Interventional

Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

NCT00264888
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 13, 2009 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 2 Interventional

PTC124 for Cystic Fibrosis

NCT00234663
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 12, 2009 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 2 Interventional Results available

Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome

NCT02758626
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 15, 2023 · Synced Sep 3, 2026, 7:27 AM EDT
Terminated Phase 3 Interventional Results available

Study of Ataluren (PTC124) in Cystic Fibrosis

NCT02107859
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 24, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Terminated Phase 2 Interventional Results available

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

NCT00759876
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 28, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Terminated Phase 2 Interventional Results available

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

NCT00847379
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 14, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 4 Interventional Results available

PTC Study to Evaluate Ataluren in Combination With Ivacaftor

NCT03256968
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ataluren
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
6 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Mar 25, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 3 Interventional Results available

Extension Study of Ataluren (PTC124) in Cystic Fibrosis

NCT01140451
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 18, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 2 Interventional Results available

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

NCT03796637
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 4, 2022 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 3 Interventional Results available

Study of Ataluren (PTC124™) in Cystic Fibrosis

NCT00803205
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ataluren, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
238 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
21
States / cities
Birmingham, Alabama • Long Beach, California • Palo Alto, California + 16 more
Source: ClinicalTrials.gov public record
Updated May 13, 2020 · Synced Sep 3, 2026, 7:27 AM EDT
Completed No phase listed Observational

The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor

NCT01182324
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 4, 2018 · Synced Sep 3, 2026, 7:27 AM EDT
Completed Phase 2 Interventional Results available

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

NCT02819557
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
2 Years to 5 Years · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
6
States / cities
Gulf Breeze, Florida • Chicago, Illinois • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2020 · Synced Sep 3, 2026, 7:27 AM EDT