DYNE-251: Administered by IV infusion
Placebo: Administered by IV infusion
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).
| 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 |
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 the UK