AOC 1044 for Duchenne muscular dystrophy with mutations suited to exon 44 skipping
Tests AOC 1044 against placebo in children with Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: 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
Key facts
- Where
- Leiden, NetherlandsSee the site
- Phase
- Phase 3
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for boys and young men aged 7 to 16 who can walk and have Duchenne muscular dystrophy (DMD) caused by a gene change that can be treated with exon 44 skipping.
- Age
- 7 to 16 years
- Sex
- Males only
- Disease stage
- Duchenne muscular dystrophy (DMD); must be able to walk (ambulatory).
- Earlier treatment
- Must have taken a stable dose of corticosteroids (including Vamolorone) for at least 6 months before Day 1, and this must stay stable. Must not have had cell or gene therapy before.
- Biomarker or test result
- A genetic test must confirm a dystrophin gene mutation amenable to exon 44 skipping.
Main reasons for not taking part
- Previous cell or gene therapy
- Treatment with another oligonucleotide within 6 months of consent (COVID-19 RNA vaccines do not count)
- Lab values outside the range set by the protocol at Screening
- Growth hormone or testosterone taken for less than 1 month before consent
- Givinostat taken for less than 6 months before consent
Read by machine on 11 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 7 and 16 years old?
Source: NCT07587242, who can take part
Where in Netherlands
- Academisch Ziekenhuis LeidenLeiden, NetherlandsTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Avidity Biosciences, Inc.
- Trial code
- NCT07587242
- Record updated
- 10 November 2026
- Original description
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 SkippingRead full original descriptionShow less
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
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
Inclusion: 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.
Exclusion: 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 outside of the protocol specified range at Screening; Less than 1 month, for growth hormone and/or testosterone; Less than 6 months for givinostat.