Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Condition(s) studied
Investigational drug(s) / intervention(s)
DYNE-251: Administered by IV infusion
Placebo: Administered by IV infusion
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 (288 weeks).
Eligibility
Primary outcome measure(s)
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — Through study completion, up to Week 337
- Change From Baseline in Dystrophin Protein Levels in Muscle Tissue at Week 25 — Baseline, Week 25
Trial sites (30)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 | Belgium | |
| UZ Leuven | Leuven | Belgium | |
| CHR Citadelle | Liège | Belgium | |
| 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 | Ireland | |
| 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 | Spain | |
| Hospital Sant Joan de Déu Universidad de Barcelona | Barcelona | Spain | |
| Alder Hey Children's Hospital | Liverpool | Merseyside | |
| Royal Victoria Infirmary | Newcastle upon Tyne | Northumberland | |
| Leeds Teaching Hospitals NHS Trust | Leeds | West Yorkshire | |
| Bristol Childrens Hospital | Bristol | United Kingdom | |
| Great Ormond Street Hospital | London | United Kingdom |
More Dyne Therapeutics trials in Spain
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05524883 on ClinicalTrials.gov ↗ ← All trials in Spain