A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (Delpacibart Zotadirsen) for the Treatment of DMD With Gene Mutations Amenable to Exon 44 Skipping
Status: Recruiting
Location: See all (13) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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
Participation Requirements
Sex: Male
Minimum Age: 7
Maximum Age: 16
Healthy Volunteers: f
View:
• 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.
Locations
Other Locations
Belgium
UZ Leuven
RECRUITING
Leuven
Centre Hospitalier Régional de la Citadelle
RECRUITING
Liège
France
AP-HP Hospital Armand-Trousseau
RECRUITING
Paris
Germany
Universitaetsklinikum Essen
RECRUITING
Essen
Universitaetsklinikum Heidelberg
RECRUITING
Heidelberg
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
RECRUITING
München
Italy
Fondazione Serena ETS - Centro Clinico NeMO Milano
RECRUITING
Milan
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro Cuore
RECRUITING
Roma
Spain
Hospital Sant Joan de Deu
RECRUITING
Barcelona
Hospital Universitario La Paz
RECRUITING
Madrid
Hospital Viamed Santa Angela De la Cruz - FutureMeds Spain
RECRUITING
Seville
United Kingdom
Royal Victoria Infirmary
RECRUITING
Newcastle
University of Oxford and Oxford University Hospitals NHS Foundation Trust
RECRUITING
Oxford
Contact Information
Primary
Avidity Bioscience, Inc., A Novartis Company
Novartis.email@novartis.com
1-888-669-6682
Time Frame
Start Date: 2026-09-01
Estimated Completion Date: 2030-07
Participants
Target number of participants: 70
Treatments
Experimental: AOC 1044
Participants will receive AOC 1044 intravenously (IV) every 6 weeks (Q6W) during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which the same participants will continue to receive AOC 1044 IV Q6W
Placebo_comparator: Placebo
Participants will receive placebo (saline) IV Q6W during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which all participants will then receive AOC 1044 IV Q6W
Related Therapeutic Areas
Sponsors
Leads: Avidity Biosciences, Inc.