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NCT03648827 · ClinicalTrials.gov registry record · Phase 2
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
A Phase 2 study, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 20
- Enrollment target
NCT03648827 is a Phase 2 study that has completed, run by PTC Therapeutics. The registered enrollment target is 20 participants.
The verdict
NCT03648827, a Phase 2 study, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 20 participants
- Enrollment target
Study 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.
Interventions
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2018-12-21 |
| Est. Completion | 2020-10-23 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03648827
The ClinicalTrials.gov registry entry for NCT03648827 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 20 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is PTC Therapeutics, which has 52 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Ataluren is the first listed.
NCT03648827 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03648827 about?
NCT03648827 is a clinical study titled "A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)". 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, electrochem...
What is the current status of trial NCT03648827?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 20 participants. The study started on 2018-12-21. Estimated completion is 2020-10-23.
What interventions are being tested in trial NCT03648827?
The interventions under investigation include: Ataluren (DRUG).
Who is sponsoring clinical trial NCT03648827?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
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