Can a new drug help boys with duchenne muscular dystrophy build muscle protein?

NCT ID NCT05524883

Ongoing Disease control Sponsor: Dyne Therapeutics Source: ClinicalTrials.gov ↗

First seen Aug 11, 2026 · Last updated Aug 12, 2026 · Updated 1 time

Summary

This trial is testing an investigational drug called DYNE-251 in boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The goal is to see if the drug can safely help their muscles produce dystrophin, a protein that is missing in DMD. Participants receive multiple IV doses of the drug or a placebo, and researchers will measure dystrophin levels in muscle tissue and monitor for side effects.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: DYNE-251, an investigational drug given by IV infusion
What this could lead to: If successful, this could lead to a treatment that helps boys with Duchenne muscular dystrophy produce dystrophin, potentially slowing muscle decline.
What could go wrong: This is an early-stage trial, so safety and effectiveness are not yet proven. The drug may not produce enough dystrophin to make a meaningful difference, and there could be side effects.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY (DMD)

Contacts and locations

Locations

Alder Hey Children's Hospital

Liverpool, Merseyside, L12 2AP, United Kingdom
Bristol Childrens Hospital

Bristol, BS2 8BJ, United Kingdom
CHI [Children's Health Ireland] at Temple Street Children's University Hospital

Dublin, D01 XD99, Ireland
CHR Citadelle

Liège, 4000, Belgium
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital at Westmead

Westmead, New South Wales, 02145, Australia
Children's Hospital of Eastern Ontario

Ottawa, Ontario, ON K1H 8L1, Canada
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Fondazione Policlinico Universitario A Gemelli

Rome, Lazio, 00168, Italy
Fondazione Serena Onlus - Centro Clinico NeMO

Milan, Lombardy, 20162, Italy
Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom
Hospital Sant Joan de Déu Universidad de Barcelona

Barcelona, 8950, Spain
Hospital Universitario Vall d'Hebron - PPDS

Barcelona, 8025, Spain
Leeds Teaching Hospitals NHS Trust

Leeds, West Yorkshire, LS1 3EX, United Kingdom
London Health Sciences Centre

London, Ontario, N6A 5W9, Canada
Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Ospedale San Raffaele S.r.l. - PPDS

Milan, Lombardy, 20312, Italy
Rare Disease Research, LLC

Atlanta, Georgia, 30329, United States
Royal Victoria Infirmary

Newcastle upon Tyne, Northumberland, NE1 4LP, United Kingdom
Samsung Medical Center

Seoul, Teugbyeolsi, 6351, South Korea
Shriners Hospitals for Children Portland

Portland, Oregon, 97239, United States
UCLA University California of Los Angeles

Los Angeles, California, 90095, United States
UMass Memorial Medical Center

Worcester, Massachusetts, 01655, United States
UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224-1334, United States
UZ Gent

Ghent, 9000, Belgium
UZ Leuven

Leuven, 3000, Belgium
University of California San Diego

La Jolla, California, 92037, United States
University of Utah - PPDS

Salt Lake City, Utah, 08412, United States
Virginia Commonwealth University

Richmond, Virginia, 23219, United States

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