NCT07587242 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
| NCT ID | NCT07587242 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Avidity Biosciences, Inc. |
| Condition | Muscular Dystrophies |
| Study Type | INTERVENTIONAL |
| Enrollment | 70 participants |
| Start Date | 2026-09-01 |
| Primary Completion | 2029-05 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 70 participants in total. It began in 2026-09-01 with a primary completion date of 2029-05.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
Eligibility Criteria
Key Inclusion Criteria: * 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. Key Exclusion Criteria: * Previous treatment cell or gene therapy. * Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines). * Lab values outside of the protocol specified range at Screening * 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: * Less than 1 month, for growth hormone and/or testosterone * Less than 6 months for givinostat
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07587242 clinical trial?
This trial is open to male participants only, aged 7 Years or older, up to 16 Years, studying Muscular Dystrophies. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07587242 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 70 participants.
Is NCT07587242 currently recruiting?
Yes, NCT07587242 is actively recruiting participants. Contact the research team at Novartis.email@novartis.com for enrollment information.
Where is the NCT07587242 trial being conducted?
This trial is being conducted at Westmead, Australia, Leuven, Belgium, Liège, Belgium, London, Canada and 11 additional locations.
Who is sponsoring the NCT07587242 clinical trial?
NCT07587242 is sponsored by Avidity Biosciences, Inc.. The trial plans to enroll 70 participants.