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
- Genova, Milan, and Roma, ItalySee the 4 sites
- Phase
- Phase 3
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 Italy
- Istituto Giannina GasliniGenova, ItalyTaking people
- Fondazione Serena ETS - Centro Clinico NeMO MilanoMilan, ItalyTaking people
- IRCCS Ospedale San RaffaeleMilan, ItalyTaking people
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro CuoreRoma, ItalyTaking 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.