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

August 31, 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

Status

Indicates the current recruitment status or the expanded access status.
Available

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Expanded Access

Expanded Access Type

Expanded Access Type

Describes the category of expanded access under U.S. Food and Drug Administration (FDA) regulations. There are three types of expanded access:
  • Individual Patients: Allows a single patient, with a serious disease or condition who cannot participate in a clinical trial, access to a drug or biological product that has not been approved by the FDA. This category also includes access in an emergency situation.
  • Intermediate-size Population: Allows more than one patient (but generally fewer patients than through a Treatment IND/Protocol) access to a drug or biological product that has not been approved by the FDA. This type of expanded access is used when multiple patients with the same disease or condition seek access to a specific drug or biological product that has not been approved by the FDA.
  • Treatment IND/Protocol: Allows a large, widespread population access to a drug or biological product that has not been approved by the FDA. This type of expanded access can only be provided if the product is already being developed for marketing for the same use as the expanded access use.
  • 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 Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

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
    • California
      • Irvine, California, United States, 92612
        • Available
        • UCI Alpha Clinic
      • San Carlos, California, United States, 94070
        • Available
        • Lucille Packard Children's Hospital at Stanford
      • San Diego, California, United States, 92121
        • Available
        • UC San Diego Health
    • Connecticut
      • Hartford, Connecticut, United States, 06106
        • Available
        • Connecticut Children's Medical Center
    • Florida
      • Kissimmee, Florida, United States, 34746
        • Available
        • Rare Disease Research
      • Miami, Florida, United States, 33155
        • Available
        • Nicklaus Children's Hospital
    • 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
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Available
        • Washington University
    • New Jersey
      • Iselin, New Jersey, United States, 08830
        • Available
        • Rare Disease Research
    • North Carolina
      • Hillsborough, North Carolina, United States, 27278
        • Available
        • Rare Disease Research
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Available
        • Nationwide Children's Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Available
        • University of Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Available
        • Children's Hospital of Philadelphia
    • South Carolina
      • Columbia, South Carolina, United States, 29203
        • Available
        • Prisma Health-Midlands Children's Hospital
    • Texas
      • Flower Mound, Texas, United States, 75028
        • Available
        • Neurology & Neuromuscular Care Center
    • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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 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

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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Avidity Biosciences, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
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

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 18, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 18, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 26, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 1, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 31, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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