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Results for “Nonsense Mutation”

52 trials

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Showing 20 of 52 results

Large-scale testing (Phase 3)Ended earlyNCT02090959
What this trial is testing

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

Who this might be right for
Muscular Dystrophy, DuchenneMuscular DystrophiesMuscular Disorders, Atrophic+6 more
PTC Therapeutics 219
Testing effectiveness (Phase 2)UnknownNCT04140786
What this trial is testing

Optimizing IV Gentamicin in JEB

Who this might be right for
Junctional Epidermolysis Bullosa
University of Southern California 6
Testing effectiveness (Phase 2)Looking for participantsNCT03526159
What this trial is testing

Gentamicin for Junctional Epidermolysis Bullosa

Who this might be right for
Junctional Epidermolysis Bullosa
University of Southern California 6
Testing effectiveness (Phase 2)Study completedNCT04336826
What this trial is testing

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

Who this might be right for
Nonsene Mutation Duchenne Muscular Dystrophy
PTC Therapeutics 6
Testing effectiveness (Phase 2)Ended earlyNCT04069260
What this trial is testing

ELX-02 in Patients With Nephropathic Cystinosis

Who this might be right for
Genetic DiseaseNonsense MutationCystinosis
Eloxx Pharmaceuticals, Inc. 3
Early research (Phase 1)Ended earlyNCT02807961
What this trial is testing

Safety, Tolerability, Pharmacokinetics of ELX-02 in Healthy Adult Volunteers

Who this might be right for
Genetic DiseasesNonsense Mutations
Eloxx Pharmaceuticals, Inc. 42
Large-scale testing (Phase 3)Study completedNCT01557400
What this trial is testing

Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

Who this might be right for
Duchenne Muscular DystrophyBecker Muscular DystrophyDystrophinopathy
PTC Therapeutics 94
Testing effectiveness (Phase 2)Study completedNCT00458341
What this trial is testing

Ataluren in Pediatric Participants With Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 30
Testing effectiveness (Phase 2)Study completedNCT02698735
What this trial is testing

Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients

Who this might be right for
Recessive Dystrophic Epidermolysis Bullosa
University of Southern California 5
Large-scale testing (Phase 3)Study completedNCT01826487
What this trial is testing

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

Who this might be right for
Muscular Dystrophy, DuchenneMuscular DystrophiesMuscular Disorders, Atrophic+6 more
PTC Therapeutics 230
Post-approval studies (Phase 4)Study completedNCT03256799
What this trial is testing

Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations

Who this might be right for
Cystic Fibrosis
University of Alabama at Birmingham 1
Testing effectiveness (Phase 2)Study completedNCT02647359
What this trial is testing

Study of Ataluren in Participants With Nonsense Mutation Aniridia

Who this might be right for
Aniridia
PTC Therapeutics 39
Post-approval studies (Phase 4)Study completedNCT03256968
What this trial is testing

PTC Study to Evaluate Ataluren in Combination With Ivacaftor

Who this might be right for
Cystic Fibrosis
University of Alabama at Birmingham 1
Large-scale testing (Phase 3)Study completedNCT01140451
What this trial is testing

Extension Study of Ataluren (PTC124) in Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 191
Testing effectiveness (Phase 2)WithdrawnNCT04117880
What this trial is testing

Open Label Extension Study in Participants With Nonsense Mutation Aniridia

Who this might be right for
Aniridia
PTC Therapeutics
Testing effectiveness (Phase 2)Study completedNCT03648827
What this trial is testing

Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 20
Large-scale testing (Phase 3)Study completedNCT02139306
What this trial is testing

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

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 279
Testing effectiveness (Phase 2)Study completedNCT00351078
What this trial is testing

PTC124 for the Treatment of Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 19
Large-scale testing (Phase 3)Study completedNCT03179631
What this trial is testing

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, DuchenneMuscular DystrophiesMuscular Disorders, Atrophic+6 more
PTC Therapeutics 360
Testing effectiveness (Phase 2)Study completedNCT00264888
What this trial is testing

Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 38
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