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

NCT07250737 | Unknown

• 1 other identifier: AOC 1044-MAP
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 17

Brief Summary

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.

Conditions

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

Keywords

Avidity; Avidity Biosciences; AOC 1044; AOC 1044-CS1; AOC 1044-CS2; EXPLORE44; EXPLORE44-OLE; Del-zota; delpacibart zotadirsen; DMD; exon skipping therapy; dystrophin; managed access; expanded access; pre-approval access; compassionate use; MAP; EAP

Interventions

delpacibart zotadirsen DRUG

Del-zota is administered as an IV infusion every 6 weeks. Doses are administered based on body weight.

Also known as: Del-zota, AOC 1044

Eligibility Criteria

• Age: 2 Years+
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Rollover Participants
• Completed Study EXPLORE44-OLE Treatment Period (through W102)
• No significant tolerability issues with AOC 1044
• New (Non-Rollover) Participants
• Permanently residing in the US and have a US primary health care provider
• Documented dystrophin gene mutation that is amenable to exon 44 skipping
• Age 2 or older at the time of consent
• If previously treated with gene therapy for DMD, treatment and associated immunosuppressive regimen was more than 12 months before consent and in the opinion of the prescriber, participant has had an unsatisfactory treatment response

You may not qualify if:

• Rollover Participants
• Prescence of any new condition or worsening of existing condition that could affect participant's safety or ability to comply with the program requirements
• New (Non-Rollover) Participants
• Recently treated with or on a clinical study for another investigation drug
• Serious respiratory or cardiac dysfunction, or nearing end of life
• Screening laboratory parameters do not meet protocol requirements
• History of multiple drug allergies or to any component of AOC 1044
• Participants who discontinued early from the treatment period of EXPLORE44 or EXPLORE44-OLE

Sponsors & Collaborators

• Avidity Biosciences, Inc.: lead
• Clinigen Healthcare Limited: collaborator

Study Sites (17)

University of Alabama

Birmingham, Alabama, 35294, United States

AVAILABLE

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

AVAILABLE

UCI Alpha Clinic

Irvine, California, 92612, United States

AVAILABLE

Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States

AVAILABLE

Rare Disease Research

Kissimmee, Florida, 34746, United States

AVAILABLE

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

AVAILABLE

Rare Disease Research

Atlanta, Georgia, 30329, United States

AVAILABLE

University of Kansas Medical Center

Kansas City, Kansas, 66103, United States

AVAILABLE

UMass Memorial Health Care

Worcester, Massachusetts, 01583, United States

AVAILABLE

Gillette Children's

Saint Paul, Minnesota, 55101, United States

AVAILABLE

Washington University

St Louis, Missouri, 63110, United States

AVAILABLE

Rare Disease Research

Iselin, New Jersey, 08830, United States

AVAILABLE

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

AVAILABLE

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

AVAILABLE

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

AVAILABLE

Prisma Health-Midlands Children's Hospital

Columbia, South Carolina, 29203, United States

AVAILABLE

University of Vermont Medical Center

Burlington, Vermont, 05401, United States

AVAILABLE

MeSH Terms

Conditions

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

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Clinigen Customer Service

CONTACT

1 877 768 4303; usmapcoordinators@clinigengroup.com

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 18, 2025
• First Posted: November 26, 2025
• Last Updated: July 30, 2026

Record last verified: 2026-04

Locations

US (17)