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

NCT ID NCT05524883

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

86 people

The number who actually took part.

Started

Aug 2022

Expected to finish

Sep 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 to 16 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Age 4 to 16 years inclusive, at the time of informed consent/assent. * Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping. * Upper extremity muscle group that is amenable to muscle biopsy. * Brooke Upper Extremity Scale score of 1 or 2. * Ambulatory or non-ambulatory. A non-ambulatory participant must have been non-ambulatory for \<2 years before enrollment. * Receiving a stable dosage of glucocorticoids for at least 12 weeks prior to the start of study drug administration, with the expectation of maintaining a stable dose during the Placebo-Controlled and Open-Label Periods of the study (unless dose adjustment is required by weight change). * Left ventricular ejection fraction of ≥50% by echocardiogram or ≥55% by cardiac magnetic resonance imaging (MRI). Exclusion Criteria: * Uncontrolled clinical symptoms and signs of congestive heart failure (CHF). * Any change in prophylaxis/treatment for CHF within 3 months prior to the start of study treatment. * History of major surgical procedure within 12 weeks prior to the start of study drug administration or an expectation of a major surgical procedure during the study. * Requirement of daytime ventilator assistance. * Percent predicted FVC \<40 % (applies only for participants who are age ≥7 years). * Receipt of eteplirsen, or alternative exon-skipping/dystrophin-modifying therapy, within 12 weeks of randomization. * Receipt of non-exon skipping investigational drug within 4 months before the start of study drug administration. * Receipt of gene therapy at any time. Other inclusion and exclusion criteria may apply.

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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

More trials for these conditions

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