Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (NCT05524883)
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
🩺 Plain-English Summary (8th-Grade Level)
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 (288 weeks).
Who can join: Review the eligibility criteria below.
Location: University of California San Diego — La Jolla, California
Age range: 4 Years to 16 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (30 Sites)
Showing the first 25 of 30 sites.
- University of California San Diego — La Jolla, California
- UCLA University California of Los Angeles — Los Angeles, California
- Children's Hospital Colorado — Aurora, Colorado
- Rare Disease Research, LLC — Atlanta, Georgia
- UMass Memorial Medical Center — Worcester, Massachusetts
- Nationwide Children's Hospital — Columbus, Ohio
- Shriners Hospitals for Children Portland — Portland, Oregon
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania
- University of Utah - PPDS — Salt Lake City, Utah
- Virginia Commonwealth University — Richmond, Virginia
- Children's Hospital at Westmead — Westmead, New South Wales
- Murdoch Children's Research Institute — Parkville, Victoria
- UZ Gent — Ghent,
- UZ Leuven — Leuven,
- CHR Citadelle — Liège,
- London Health Sciences Centre — London, Ontario
- Children's Hospital of Eastern Ontario — Ottawa, Ontario
- CHI [Children's Health Ireland] at Temple Street Children's University Hospital — Dublin,
- Fondazione Policlinico Universitario A Gemelli — Rome, Lazio
- Fondazione Serena Onlus - Centro Clinico NeMO — Milan, Lombardy
- Ospedale San Raffaele S.r.l. - PPDS — Milan, Lombardy
- Samsung Medical Center — Seoul, Teugbyeolsi
- Hospital Universitario Vall d'Hebron - PPDS — Barcelona,
- Hospital Sant Joan de Déu Universidad de Barcelona — Barcelona,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Duchenne Muscular Dystrophy (DMD)
- Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy — Nashville, Tennessee
- The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy — Rochester, New York
- Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) — Hillsborough, North Carolina
- Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials — Sacramento, California
- Duchenne Electronic Health Record Study — Little Rock, Arkansas
Explore by condition: All Duchenne Muscular Dystrophy (DMD) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.