Trials / Terminated
TerminatedNCT02090959
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
- Status
- Terminated
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 219 (actual)
- Sponsor
- PTC Therapeutics · Industry
- Sex
- Male
- Age
- 7 Years – 15 Years
- Healthy volunteers
- Not accepted
Summary
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren \[PTC124-GD-020-DMD {NCT01826487}\]) to augment the overall safety database. Screening and baseline procedures are structured to avoid a gap in treatment between the double-blind study (PTC124-GD-020-DMD) and this extension study. This study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is discontinued.
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
| Type | Name | Description |
|---|---|---|
| DRUG | Ataluren | Ataluren will be administered per the dose and schedule specified in the arm. |
Timeline
- Start date
- 2014-03-20
- Primary completion
- 2018-06-12
- Completion
- 2018-06-12
- First posted
- 2014-03-19
- Last updated
- 2020-08-11
- Results posted
- 2020-08-11
Locations
58 sites across 19 countries: United States, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, Czechia, France, Germany, Israel, Italy, Poland, South Korea, Spain, Sweden, Switzerland, Turkey (Türkiye), United Kingdom
Source: ClinicalTrials.gov record NCT02090959. Inclusion in this directory is not an endorsement.