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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: Completed Phase 2 study, sponsored by PTC Therapeutics.
NCT03648827 is a Phase 2 study that has completed, run by PTC Therapeutics. The registered enrollment target is 20 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (85% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
The percent change in dystrophin level from baseline in ambulatory nonsense mutation duchenne muscular dystrophy (nmDMD) participants after treatment with ataluren for 40 weeks was analyzed using quantitative assay (ECL).The least square (LS) mean and 90% confidence interval (CI) were analyzed from a mixed-model repeated measures (MMRM) with factors of muscle locations and visits as fixed effects, and participants as a random effect.
Interventions
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2018-12-21 |
| Est. Completion | 2020-10-23 |
| Phase | Phase 2 |
What the finished NCT03648827 record still lists
NCT03648827 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 20 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (85% lower).
The record links to 0 conditions, and to 1 intervention - of which Ataluren is the first listed.
NCT03648827 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03648827's enrollment target sits among peer trials
20 1805th of 2000 higher than 107 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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