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

May 21, 2026 updated by: Avidity Biosciences, Inc.

Managed Access to Investigational Use of AOC 1044 in Participants With DMD Mutations Amenable to Exon 44 Skipping

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.

Study Overview

Status

Available

Conditions

Intervention / Treatment

Study Type

Expanded Access

Expanded Access Type

  • Treatment IND/Protocol

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • Available
        • University of Alabama
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Available
        • Arkansas Children's Hospital
    • Florida
      • Kissimmee, Florida, United States, 34746
        • Available
        • Rare Disease Research
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Available
        • Rare Disease Research
    • Kansas
      • Kansas City, Kansas, United States, 66103
        • Available
        • University of Kansas Medical Center
    • Massachusetts
      • Worcester, Massachusetts, United States, 01583
        • Available
        • UMass Memorial Health Care
    • Minnesota
      • Saint Paul, Minnesota, United States, 55101
        • Available
        • Gillette Children's
    • New Jersey
      • Iselin, New Jersey, United States, 08830
        • Available
        • Rare Disease Research
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Available
        • Nationwide Children's Hospital
    • South Carolina
      • Columbia, South Carolina, United States, 29203
        • Available
        • Prisma Health-Midlands Children's Hospital
    • Vermont
      • Burlington, Vermont, United States, 05401
        • Available
        • University of Vermont Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

Key Inclusion Criteria

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 6 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

Key Exclusion Criteria

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Avidity Biosciences, Inc.

Collaborators

Clinigen Healthcare Limited

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

November 18, 2025

First Submitted That Met QC Criteria

November 18, 2025

First Posted (Actual)

November 26, 2025

Study Record Updates

Last Update Posted (Actual)

May 26, 2026

Last Update Submitted That Met QC Criteria

May 21, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AOC 1044-MAP

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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