Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping

Available (expanded access)

Conditions studied: Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Muscular Dystrophies, Muscular Dystrophy, Duchenne

In brief

The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Key facts

Study ID

NCT07250737

Run by

Avidity Biosciences, Inc.

Last updated by the study team

2026-07-30

Who can join

Age: 2 and older. Sex: male. Healthy volunteers: not accepted.

Where it is running

• University of Alabama — Birmingham, Alabama, United States
• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• UCI Alpha Clinic — Irvine, California, United States
• Connecticut Children's Medical Center — Hartford, Connecticut, United States
• Rare Disease Research — Kissimmee, Florida, United States
• Nicklaus Children's Hospital — Miami, Florida, United States
• Rare Disease Research — Atlanta, Georgia, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• UMass Memorial Health Care — Worcester, Massachusetts, United States
• Gillette Children's — Saint Paul, Minnesota, United States
• Washington University — St Louis, Missouri, United States
• Rare Disease Research — Iselin, New Jersey, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• Prisma Health-Midlands Children's Hospital — Columbia, South Carolina, United States
• University of Vermont Medical Center — Burlington, Vermont, United States

Full record on ClinicalTrials.gov

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