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
AOC 1044: AOC 1044 will be administered by intravenous (IV) infusion
Placebo: Placebo will be administered by intravenous (IV) infusion
Study 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
Sex
MALE
Min age
7 Years
Max age
16 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Change from Baseline in Time to Rise (TTR) Velocity at Week 54 — Baseline, Week 54
Trial sites (14)
Facility
City
Region
Status
UZ Leuven
Leuven
Belgium
Recruiting
Centre Hospitalier Régional de la Citadelle
Liège
Belgium
Recruiting
AP-HP Hospital Armand-Trousseau
Paris
France
Recruiting
Universitaetsklinikum Essen
Essen
Germany
Recruiting
Universitaetsklinikum Heidelberg
Heidelberg
Germany
Recruiting
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
München
Germany
Recruiting
Istituto Giannina Gaslini
Genova
Italy
Recruiting
Fondazione Serena ETS - Centro Clinico NeMO Milano
Milan
Italy
Recruiting
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro Cuore
Roma
Italy
Recruiting
Hospital Sant Joan de Deu
Barcelona
Spain
Recruiting
Hospital Universitario La Paz
Madrid
Spain
Recruiting
Hospital Viamed Santa Angela De la Cruz - FutureMeds Spain
Seville
Spain
Recruiting
Royal Victoria Infirmary
Newcastle
United Kingdom
Recruiting
University of Oxford and Oxford University Hospitals NHS Foundation Trust
Oxford
United Kingdom
Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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