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Results for “Nonsense Mutation”

52 trials

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Showing 20 of 52 results

Testing effectiveness (Phase 2)Study completedNCT00234663
What this trial is testing

PTC124 for Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 24
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07523581
What this trial is testing

EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety

Who this might be right for
Alport Syndrome, X-LinkedAlport Syndrome, Autosomal Recessive
Eloxx Pharmaceuticals, Inc. 24
Testing effectiveness (Phase 2)Study completedNCT02819557
What this trial is testing

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

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 14
Large-scale testing (Phase 3)Study completedNCT01247207
What this trial is testing

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

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 270
Testing effectiveness (Phase 2)Study completedNCT05448755
What this trial is testing

ELX-02 in Patients With Alport Syndrome

Who this might be right for
Alport Syndrome
Eloxx Pharmaceuticals, Inc. 3
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)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
Large-scale testing (Phase 3)Ended earlyNCT02456103
What this trial is testing

Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 246
Early research (Phase 1)Study completedNCT03292302
What this trial is testing

Phase 1 Study of ELX-02 in Healthy Adults

Who this might be right for
Genetic DiseaseNonsense Mutation
Eloxx Pharmaceuticals, Inc. 18
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
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
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
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
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 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)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 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 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)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
Post-approval studies (Phase 4)UnknownNCT04454151
What this trial is testing

Azithromycin Treatment for Readthrough of APC Gene Stop Codon Mutations in Familial Adenomatous Polyposis

Who this might be right for
Familial Adenomatous Polyposis
Tel-Aviv Sourasky Medical Center 10
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