Trials / Completed
CompletedNCT03648827
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment With Ataluren
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 20 (actual)
- Sponsor
- PTC Therapeutics · Industry
- Sex
- Male
- Age
- 2 Years – 7 Years
- Healthy volunteers
- Not accepted
Summary
This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD. The study will evaluate the levels of dystrophin before and after 40 weeks of ataluren therapy using muscle biopsies and 2 validated assay methods, electrochemiluminescence (ECL) and immunohistochemistry.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Ataluren | Ataluren will be administered as per the dose and schedule specified in the arm. |
Timeline
- Start date
- 2018-12-21
- Primary completion
- 2020-10-23
- Completion
- 2020-10-23
- First posted
- 2018-08-27
- Last updated
- 2022-04-05
- Results posted
- 2022-04-05
Locations
9 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03648827. Inclusion in this directory is not an endorsement.