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Hope for duchenne: new drug targets rare gene mutation in phase 3 trial

NCT ID NCT07587242

Disease control Sponsor: Avidity Biosciences, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Sep 16, 2026 · Updated 14 times

Summary

This study tests an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). About 70 boys aged 7 to 16 who can still walk will receive either the drug or a placebo. The main goal is to see if the drug helps them stand up faster after 54 weeks of treatment.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 70 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2026

Expected to finish

Jul 2030

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

7 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.

Key Inclusion Criteria: * Ambulatory males with clinical and genetic diagnosis of DMD * Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping * 7 to 16 years of age at time of consent * TTR and NSAA assessment completed within the protocol specified parameters at Screening * On a stable regimen of corticosteroids (including Vamolorone) for at least 6 months prior to Day 1. Steroid regimen must be anticipated to remain stable. Key Exclusion Criteria: * Previous treatment cell or gene therapy. * Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines). * Lab values outside of the protocol specified range at Screening * If on any of the following treatments (growth hormone, testosterone or givinostat), participants must be on a stable regimen and must plan to maintain it for the duration of the study. Participants will be excluded if regimen stability prior to informed consent is as follows: * Less than 1 month, for growth hormone and/or testosterone * Less than 6 months for givinostat

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Conditions

The condition(s) this trial relates to.

atrophic muscular disease congenital Duchenne muscular dystrophy hereditary disease Infant, Newborn, Diseases inherited muscle tissue disorder Muscular Disorders, Atrophic muscular dystrophy musculoskeletal system disorder myopathy myotonic dystrophy nervous system disorder neuromuscular disease perinatal disease X-linked disease

As listed by the trial registrant

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

Congenital DMD Duchene muscular dystrophy Genetic diseases Hereditary Muscular disease Muscular disorders, atrophic Muscular dystrophies Muscular dystrophies (Duchenne, Becker, myotonic dystrophy) Musculoskeletal diseases Neonatal disease Nervous system diseases Neuromuscular diseases (NMD) X-linked

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    14 sites in 6 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • AP-HP Hospital Armand-Trousseau

    RECRUITING

    Paris, France

  • Centre Hospitalier Régional de la Citadelle

    RECRUITING

    Liège, Belgium

  • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro Cuore

    RECRUITING

    Roma, Italy

  • Fondazione Serena ETS - Centro Clinico NeMO Milano

    RECRUITING

    Milan, Italy

  • Hospital Sant Joan de Deu

    RECRUITING

    Barcelona, Spain

  • Hospital Universitario La Paz

    RECRUITING

    Madrid, Spain

  • Hospital Viamed Santa Angela De la Cruz - FutureMeds Spain

    RECRUITING

    Seville, Spain

  • Istituto Giannina Gaslini

    RECRUITING

    Genova, Italy

  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

    RECRUITING

    München, Germany

  • Royal Victoria Infirmary

    RECRUITING

    Newcastle, United Kingdom

  • UZ Leuven

    RECRUITING

    Leuven, Belgium

  • Universitaetsklinikum Essen

    RECRUITING

    Essen, Germany

  • Universitaetsklinikum Heidelberg

    RECRUITING

    Heidelberg, Germany

  • University of Oxford and Oxford University Hospitals NHS Foundation Trust

    RECRUITING

    Oxford, United Kingdom

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