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Results for “Nonsense Mutation”

51 trials

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Showing 20 of 51 results

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 completedNCT00237380
What this trial is testing

Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
PTC Therapeutics 24
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
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 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 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
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)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 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)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)UnknownNCT05448755
What this trial is testing

ELX-02 in Patients With Alport Syndrome

Who this might be right for
Alport Syndrome
Eloxx Pharmaceuticals, Inc. 8
Post-approval studies (Phase 4)UnknownNCT02175914
What this trial is testing

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

Who this might be right for
FAP-Familial Adenomatous Polyposis
michal roll 20
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
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
Not applicableActive Not RecruitingNCT02369731
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 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
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
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
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
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