NCT07587242RecruitingPhase 3
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
Source: ClinicalTrials.gov · Last updated 13 Oct 2026
Brief summary (registry, in English)
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
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 70 (estimated)
- First submitted
- 8 May 2026
- Start date
- Sept 2026
- Primary completion date
- May 2029
- Lead sponsor
- Avidity Biosciences, Inc.
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT07587242
What you commit to
- Study type: Interventional · Phase 3
- Planned participants: 70
- From Sept 2026 to the primary completion date, May 2029
- 18 sites in 8 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Previous treatment cell or gene therapy.
- Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines).
- 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:
Lab values
- Lab values outside of the protocol specified range at Screening
Other
- Less than 1 month, for growth hormone and/or testosterone
- Less than 6 months for givinostat
Who the study is looking for
- 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.
Age in the registry: 7 years to 16 years · Sex: Male · Does not accept healthy volunteers
Sites (18)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| The Children's Hospital at Westmead Coordinator of this site? Confirm its status | Westmead | Australia | Recruiting |
| UZ Leuven Coordinator of this site? Confirm its status | Leuven | Belgium | Recruiting |
| Centre Hospitalier Regional de la Citadelle Coordinator of this site? Confirm its status | Liège | Belgium | Recruiting |
| Universitaetsklinikum - Essen Coordinator of this site? Confirm its status | Essen | Germany | Recruiting |
| Universitaetsklinikum Heidelberg Coordinator of this site? Confirm its status | Heidelberg | Germany | Recruiting |
| Klinikum der Ludwig-Maximilians-Universitaet Muenchen Coordinator of this site? Confirm its status | München | Germany | Recruiting |
| Hospital Sant Joan de Déu Coordinator of this site? Confirm its status | Barcelona | Spain | Recruiting |
| Hospital Universitario La Paz Coordinator of this site? Confirm its status | Madrid | Spain | Recruiting |
| Hospital Viamed Santa Angela De la Cruz - FutureMeds Spain Coordinator of this site? Confirm its status | Seville | Spain | Recruiting |
| CHU de Nantes - Hôtel Dieu Coordinator of this site? Confirm its status | Nantes | France | Recruiting |
| AP-HP Hospital Armand-Trousseau Coordinator of this site? Confirm its status | Paris | France | Recruiting |
| Royal Victoria Infirmary Coordinator of this site? Confirm its status | Newcastle | United Kingdom | Recruiting |
| University of Oxford and Oxford University Hospitals NHS Foundation Trust Coordinator of this site? Confirm its status | Oxford | United Kingdom | Recruiting |
| Istituto Giannina Gaslini Coordinator of this site? Confirm its status | Genova | Italy | Recruiting |
| Fondazione Serena ETS - Centro Clinico NeMO Milano Coordinator of this site? Confirm its status | Milan | Italy | Recruiting |
| IRCCS Ospedale San Raffaele Coordinator of this site? Confirm its status | Milan | Italy | Recruiting |
| Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro Cuore Coordinator of this site? Confirm its status | Roma | Italy | Recruiting |
| Academisch Ziekenhuis Leiden Coordinator of this site? Confirm its status | Leiden | Netherlands | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.