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
Key facts
- Where
- Genoa, Milan és Rome, OlaszországSee the 4 sites
- Phase
- 3. fázis
Where in Olaszország
- IRCCS Istituto Giannina GasliniGenoa, OlaszországTaking people
- Centro Clinico NemoMilan, OlaszországTaking people
- Ospedale San Raffaele S.r.l.Milan, OlaszországTaking people
- Fondazione Policlinico Universitario Agostino Gemelli IRCCSRome, OlaszországTaking 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
- EU 2025-523087-20-00
- Record updated
- 2026. május 27.
- Original description
To evaluate the efficacy of AOC 1044 on muscle functionRead full original descriptionShow less
To evaluate the efficacy of AOC 1044 on muscle function
Inclusion: Participant and participant’s legally designated representative have given written informed consent and/or assent (signed and dated); Ambulatory males with a documented clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years; Acceptable genetic test confirming out-of-frame dystrophin gene mutation amenable to exon 44 skipping; 7 to 16 years of age (inclusive) at the time of informed consent; Participants must be on a stable regimen of corticosteroids for at least 6 months prior to Day 1. The regimen must be anticipated to remain stable for the duration of the study.
Exclusion: Previous treatment with a cell or gene therapy product; Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines); If on any of the following treatments, participants must be on a stable regimen and must plan to maintain the regimen for the duration of the study (appropriate changes to account for body weight and composition). Participants will be excluded if regimen stability prior to informed consent is as follows: a. For growth hormone and/or testosterone: <1 month. b. For givinostat: <6 months.