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Clinical Trials in the UK / NCT07587242
Recruiting Phase 3

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

NCT07587242 · tracked via the Priya Life Science UK tracker
Sponsor
Avidity Biosciences, Inc.
Phase
Phase 3
Started
2026-09-01
Last updated
2026-09-15

Condition(s) studied

Muscular DystrophiesMuscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)Muscular Disorders, AtrophicMuscular DiseaseMusculoskeletal DiseasesNeuromuscular Diseases (NMD)Nervous System DiseasesGenetic DiseasesX-LinkedHereditaryNeonatal DiseaseDuchene Muscular DystrophyCongenitalDMD

Investigational drug(s) / intervention(s)

AOC 1044Placebo

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)

Trial sites (14)

FacilityCityRegionStatus
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.

View NCT07587242 on ClinicalTrials.gov ↗ ← All trials in the UK