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 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: AOC 1044-CS3
• Study Type: interventional
• Target: 70
• Locations: 7 countries
• Sites: 17

Brief 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

Conditions

Genetic Diseases; Muscular Disorders, Atrophic; Duchene Muscular Dystrophy; Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy); Muscular Disease; Musculoskeletal Diseases; Neuromuscular Diseases (NMD); Nervous System Diseases; Neonatal Disease; Congenital; Muscular Dystrophies; X-Linked; Hereditary; DMD

Keywords

AOC; AOC 1044; AOC 1044-CS3; AOC 1044-CS1; AOC 1044-CS2; EXPLORE44; EXPLORE44-OLE; SAFARI; SAFARI44; SAFARI 44; Avidity; Avidity Biosciences; Exon Skipping Therapy; Avidity Biosciences Inc., A Novartis Company; del-zota; DMD

Outcome Measures

Primary Outcomes (1)

• Change from Baseline in Time to Rise (TTR) Velocity at Week 54

  Baseline, Week 54

Secondary Outcomes (11)

• Change from Baseline in Creatine Kinase (CK) at Week 54

  Baseline, Week 54

• Change from Baseline in 4-Stair Climb (4SC) Velocity at Week 54

  Baseline, Week 54

• Change from Baseline in 10-Meter Walk/Run Test (10MWRT) Velocity at Week 54

  Baseline, Week 54

• Change from Baseline in Stride Velocity 95th Centile (SV95C) at Week 54

  Baseline, Week 54

• Change from Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 54

  Baseline, Week 54

Study Arms (2)

AOC 1044

EXPERIMENTAL

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

Drug: AOC 1044

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

AOC 1044 DRUG

AOC 1044 will be administered by intravenous (IV) infusion

AOC 1044

Placebo DRUG

Placebo will be administered by intravenous (IV) infusion

Placebo

Eligibility Criteria

• Age: 7 Years - 16 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Ambulatory males with clinical and genetic diagnosis of DMD
• Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping
• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Avidity Biosciences, Inc.: lead

Study Sites (17)

UZ Leuven

Leuven, Belgium

RECRUITING

Centre Hospitalier Régional de la Citadelle

Liège, Belgium

RECRUITING

CHU de Nantes - Hotel-Dieu

Nantes, France

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

IRCCS Ospedale San Raffaele

Milan, Italy

RECRUITING

Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro Cuore

Roma, Italy

RECRUITING

Academisch Ziekenhuis Leiden

Leiden, Netherlands

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

Related Links

• Related Info

MeSH Terms

Conditions

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Myotonic Dystrophy; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Infant, Newborn, Diseases

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases

Central Study Contacts

Avidity Bioscience, Inc., A Novartis Company

CONTACT

1-888-669-6682; Novartis.email@novartis.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2026
• First Posted: May 14, 2026
• Study Start: September 1, 2026
• Primary Completion (Estimated): May 1, 2029
• Study Completion (Estimated): July 1, 2030
• Last Updated: September 29, 2026

Record last verified: 2026-08

Locations

DE (3); ES (3); FR (2); IT (4); GB (2); BE (2); NL (1)