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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

RecruitingPhase 3Ages 7 Years - 16 YearsMaleAvidity Biosciences, Inc.
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In plain words

What the trial is, and whether it could suit you

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

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📋 Eligibility rules (as published)
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
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What it tests

  • AOC 1044
  • Placebo
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Where

18 sites
The Children's Hospital at Westmead
Westmead, Australia
UZ Leuven
Leuven, Belgium
Centre Hospitalier Régional de la Citadelle
Liège, Belgium
CHU de Nantes - Hotel-Dieu
Nantes, France
AP-HP Hospital Armand-Trousseau
Paris, France
Universitaetsklinikum Essen
Essen, Germany
Universitaetsklinikum Heidelberg
Heidelberg, Germany
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
München, Germany

…and 10 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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