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Results for “Nonsense Mutations”

52 trials

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Showing 20 of 52 results

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
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
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
Not applicableStudy completedNCT02369731
What this trial is testing

Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Who this might be right for
Muscular Dystrophy, Duchenne
PTC Therapeutics 316
Large-scale testing (Phase 3)Ended earlyNCT02107859
What this trial is testing

Study of Ataluren (PTC124) in Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 61
Testing effectiveness (Phase 2)Study completedNCT02758626
What this trial is testing

Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome

Who this might be right for
Epilepsy
NYU Langone Health 15
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
Early research (Phase 1)Study completedNCT03309605
What this trial is testing

Phase 1 Study of ELX-02 in Healthy Adult Subjects

Who this might be right for
Genetic DiseaseNonsense Mutation
Eloxx Pharmaceuticals, Inc. 62
Testing effectiveness (Phase 2)Study completedNCT04126473
What this trial is testing

Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele

Who this might be right for
Cystic Fibrosis
Eloxx Pharmaceuticals, Inc. 17
Testing effectiveness (Phase 2)UnknownNCT01918384
What this trial is testing

Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, Duchenne
Kobe University 21
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
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
Not applicableLooking for participantsNCT03670472
What this trial is testing

Correction of Nonsense Mutations in Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
University Hospital, Lille 85
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
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)Ended earlyNCT00847379
What this trial is testing

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

Who this might be right for
Duchenne Muscular DystrophyBecker Muscular Dystrophy
PTC Therapeutics 173
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
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
Testing effectiveness (Phase 2)Study completedNCT00592553
What this trial is testing

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

Who this might be right for
Duchenne Muscular DystrophyBecker Muscular Dystrophy
PTC Therapeutics 174
Testing effectiveness (Phase 2)Ended earlyNCT01141075
What this trial is testing

Ataluren for Nonsense Mutation Methylmalonic Acidemia

Who this might be right for
Amino Acid Metabolism, Inborn Errors
PTC Therapeutics 11
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