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NCT05524883 · ClinicalTrials.gov registry record · Phase 1
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
A Phase 1 study of Duchenne Muscular Dystrophy (DMD), sponsored by Dyne Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 86
- Enrollment target
- 20
- Study locations
NCT05524883: Active Phase 1 study of Duchenne Muscular Dystrophy (DMD), sponsored by Dyne Therapeutics.
NCT05524883 is a Phase 1 study of Duchenne Muscular Dystrophy (DMD) that is active but no longer recruiting, run by Dyne Therapeutics. The registered enrollment target is 86 participants, above the 75-participant average among 3 other Duchenne Muscular Dystrophy (DMD) trials with a reported enrollment target (15% higher). The trial reports 20 study locations across 14 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05524883, a Phase 1 study of Duchenne Muscular Dystrophy (DMD), is active but no longer recruiting, sponsored by Dyne Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 86 participants
- Enrollment target
- 20
- Study locations
Study Summary
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (192 weeks).
Conditions Studied
Interventions
- DRUG Placebo
- DRUG DYNE-251
Study Locations (20)
Other
- UZ Gent - Ghent
- UZ Leuven - Leuven
- CHR Citadelle - Liège
- CHI [Children's Health Ireland] at Temple Street Children's University Hospital - Dublin
California
- University of California San Diego - La Jolla
- UCLA University California of Los Angeles - Los Angeles
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
- UPMC Children's Hospital of Pittsburgh - Pittsburgh
Ontario
- London Health Sciences Centre - London
- Children's Hospital of Eastern Ontario - Ottawa
Colorado
- Children's Hospital Colorado - Aurora
Georgia
- Rare Disease Research, LLC - Atlanta
Massachusetts
- UMass Memorial Medical Center - Worcester
Ohio
- Nationwide Children's Hospital - Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 86 participants |
| Start Date | 2022-08-12 |
| Est. Completion | 2029-11 |
| Phase | Phase 1 |
What the registry record for NCT05524883 still lists
NCT05524883 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 86 participants, a relatively small participant target, above the 75-participant average among 3 other Duchenne Muscular Dystrophy (DMD) trials with a reported enrollment target (15% higher).
The record links to 1 condition, with Duchenne Muscular Dystrophy (DMD) appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT05524883 names 20 study sites across 14 states, led by Other, California, Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT05524883 about?
NCT05524883 is a clinical study titled "Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping". The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a mul...
What is the current status of trial NCT05524883?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 86 participants. The study started on 2022-08-12. Estimated completion is 2029-11.
What conditions does trial NCT05524883 study?
This clinical trial studies the following conditions: Duchenne Muscular Dystrophy (DMD).
What interventions are being tested in trial NCT05524883?
The interventions under investigation include: Placebo (DRUG), DYNE-251 (DRUG).
Who is sponsoring clinical trial NCT05524883?
This trial is sponsored by Dyne Therapeutics, which has 4 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05524883 being conducted?
This trial has 20 study locations across California, Colorado, Georgia, Massachusetts, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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