AOC 1044 in Duchenne Muscular Dystrophy with Exon 44 Skipping

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 3, randomized, double-blind, placebo-controlled trial evaluating the safety and effectiveness of AOC 1044 in ambulatory males with DMD aged 7–16 who have a dystrophin mutation amenable to exon 44 skipping. Participants will receive infusions every 6 weeks for 54 weeks, followed by an open-label extension and a safety follow-up, with safety oversight by an independent Data Monitoring Committee. The study includes a screening period and requires stable corticosteroid use prior to enrollment. Key outcomes include functional and safety measures, assessed over the trial duration. Locations include Children’s Hospital at Westmead, UZ Leuven, and Centre Hospitalier Régional de la Citadelle.

Why it matters

Who can join: the full rules

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

Where it's running

• The Children's Hospital at Westmead
  Westmead, New South Wales, Australia Recruiting
• UZ Leuven
  Leuven, Belgium Recruiting
• Centre Hospitalier Régional de la Citadelle
  Liège, Belgium Recruiting
• London Health Sciences Centre - Victoria Hospital
  London, Ontario, Canada Recruiting
• CHU de Nantes - Hotel-Dieu
  Nantes, France Recruiting
• AP-HP Hospital Armand-Trousseau
  Paris, France Recruiting

Study contacts

• Study coordinator: Avidity Bioscience, Inc., A Novartis Company
• Email: Novartis.email@novartis.com
• Phone: 1-888-669-6682

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.