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Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Sponsor: Dyne Therapeutics
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).
Official title: 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
Key Details
Gender
MALE
Age Range
4 Years - 16 Years
Study Type
INTERVENTIONAL
Enrollment
86
Start Date
2022-08-12
Completion Date
2029-11
Last Updated
2025-08-13
Healthy Volunteers
No
Conditions
Interventions
DYNE-251
Administered by IV infusion
Placebo
Administered by IV infusion
Locations (30)
University of California San Diego
La Jolla, California, United States
UCLA University California of Los Angeles
Los Angeles, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
UMass Memorial Medical Center
Worcester, Massachusetts, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Shriners Hospitals for Children Portland
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Utah - PPDS
Salt Lake City, Utah, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Children's Hospital at Westmead
Westmead, New South Wales, Australia
Murdoch Children's Research Institute
Parkville, Victoria, Australia
UZ Gent
Ghent, Belgium
UZ Leuven
Leuven, Belgium
CHR Citadelle
Liège, Belgium
London Health Sciences Centre
London, Ontario, Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
CHI [Children's Health Ireland] at Temple Street Children's University Hospital
Dublin, Ireland
Fondazione Policlinico Universitario A Gemelli
Rome, Lazio, Italy
Fondazione Serena Onlus - Centro Clinico NeMO
Milan, Lombardy, Italy
Ospedale San Raffaele S.r.l. - PPDS
Milan, Lombardy, Italy
Samsung Medical Center
Seoul, Teugbyeolsi, South Korea
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, United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne, Northumberland, United Kingdom
Leeds Teaching Hospitals NHS Trust
Leeds, West Yorkshire, United Kingdom
Bristol Childrens Hospital
Bristol, United Kingdom
Great Ormond Street Hospital
London, United Kingdom