A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (Delpacibart Zotadirsen) for the Treatment of DMD With Gene Mutations Amenable to Exon 44 Skipping
- Muscular Dystrophies
- Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
- Muscular Disorders, Atrophic
- Muscular Disease
- Musculoskeletal Diseases
- Neuromuscular Diseases (NMD)
- Nervous System Diseases
- Genetic Diseases
At a glance
- Phase
- Phase 3
- Study type
- Interventional
- Sponsor
- Avidity Biosciences, Inc.
- Enrolment target
- 70
- Started
- 1 September 2026
- Main results due
- May 2029
- Study sites
- 17
- Registry updated
- 29 September 2026
Can you take part?
- Ages 7 years to 16 years.
- Men only.
- You need the condition being studied — healthy volunteers are not accepted.
These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.
Read the full eligibility criteria
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
What this study is about
In the sponsor’s own words, from the registry.
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
Study sites(17)
UZ Leuven
Leuven, Belgium
Recruiting
Centre Hospitalier Régional de la Citadelle
Liège, Belgium
Recruiting
CHU de Nantes - Hotel-Dieu
Nantes, France
Recruiting
AP-HP Hospital Armand-Trousseau
Paris, France
Recruiting
Universitaetsklinikum Essen
Essen, Germany
Recruiting
Universitaetsklinikum Heidelberg
Heidelberg, Germany
Recruiting
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
München, Germany
Recruiting
Istituto Giannina Gaslini
Genova, Italy
Recruiting
Fondazione Serena ETS - Centro Clinico NeMO Milano
Milan, Italy
Recruiting
IRCCS Ospedale San Raffaele
Milan, Italy
Recruiting
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro Cuore
Roma, Italy
Recruiting
Academisch Ziekenhuis Leiden
Leiden, Netherlands
Recruiting
Hospital Sant Joan de Deu
Barcelona, Spain
Recruiting
Hospital Universitario La Paz
Madrid, Spain
Recruiting
Hospital Viamed Santa Angela De la Cruz - FutureMeds Spain
Seville, Spain
Recruiting
Royal Victoria Infirmary
Newcastle, United Kingdom
Recruiting
University of Oxford and Oxford University Hospitals NHS Foundation Trust
Oxford, United Kingdom
Recruiting
Showing 17 of 17 sites. 17 of the 17 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.
Source: ClinicalTrials.gov record NCT07587242. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.
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