Trials / Completed
CompletedNCT03796637
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
Phase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mutation Duchenne Muscular Dystrophy Who Have Been Treated With Ataluren for ≥9 Months
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 6 (actual)
- Sponsor
- PTC Therapeutics · Industry
- Sex
- Male
- Age
- —
- Healthy volunteers
- Not accepted
Summary
This study is designed to generate additional data on the effect of ataluren for producing dystrophin protein in nonsense mutation nmDMD participants. This study will evaluate dystrophin levels from participants with nmDMD who currently have been receiving ataluren for ≥9 months. The study will have a single visit (Visit 1).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Ataluren | Ataluren will be administered as per the dose and schedule specified in the arm. |
Timeline
- Start date
- 2019-04-11
- Primary completion
- 2019-06-03
- Completion
- 2019-06-03
- First posted
- 2019-01-08
- Last updated
- 2022-04-05
- Results posted
- 2022-04-05
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03796637. Inclusion in this directory is not an endorsement.