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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.
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

Full criteria on ClinicalTrials.gov

Sites (18)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
The Children's Hospital at Westmead
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WestmeadAustraliaRecruiting
UZ Leuven
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LeuvenBelgiumRecruiting
Centre Hospitalier Regional de la Citadelle
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LiègeBelgiumRecruiting
Universitaetsklinikum - Essen
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EssenGermanyRecruiting
Universitaetsklinikum Heidelberg
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HeidelbergGermanyRecruiting
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
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MünchenGermanyRecruiting
Hospital Sant Joan de Déu
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BarcelonaSpainRecruiting
Hospital Universitario La Paz
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MadridSpainRecruiting
Hospital Viamed Santa Angela De la Cruz - FutureMeds Spain
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SevilleSpainRecruiting
CHU de Nantes - Hôtel Dieu
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NantesFranceRecruiting
AP-HP Hospital Armand-Trousseau
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ParisFranceRecruiting
Royal Victoria Infirmary
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NewcastleUnited KingdomRecruiting
University of Oxford and Oxford University Hospitals NHS Foundation Trust
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OxfordUnited KingdomRecruiting
Istituto Giannina Gaslini
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GenovaItalyRecruiting
Fondazione Serena ETS - Centro Clinico NeMO Milano
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MilanItalyRecruiting
IRCCS Ospedale San Raffaele
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MilanItalyRecruiting
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro Cuore
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RomaItalyRecruiting
Academisch Ziekenhuis Leiden
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LeidenNetherlandsRecruiting

This page is information, not medical advice. Discuss any trial with your doctor.