Muscular Disorders, AtrophicNCT07250737
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
A study testing delpacibart zotadirsen in people with Muscular Disorders, Atrophic and Muscular Diseases, run by Avidity Biosciences, Inc. at 22 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 years and older, men only
- Conditions the study is for
- Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases
- What is being tested?
- delpacibart zotadirsen
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 22 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- Men only
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
22 sites in 1 country.
22 sites in 1 country
United States22 sites
- Available
Rare Disease Research
Atlanta, Georgia, 30329
- Available
University of Alabama
Birmingham, Alabama, 35294
- Available
University of Vermont Medical Center
Burlington, Vermont, 05401
- Available
Prisma Health-Midlands Children's Hospital
Columbia, South Carolina, 29203
- Available
Nationwide Children's Hospital
Columbus, Ohio, 43205
- Available
Neurology & Neuromuscular Care Center
Flower Mound, Texas, 75028
- Available
Connecticut Children's Medical Center
Hartford, Connecticut, 06106
- Available
Rare Disease Research
Hillsborough, North Carolina, 27278
- Available
UTHealth
Houston, Texas, 77030
- Available
UCI Alpha Clinic
Irvine, California, 92612
- Available
Rare Disease Research
Iselin, New Jersey, 08830
- Available
University of Kansas Medical Center
Kansas City, Kansas, 66103
- Available
Rare Disease Research
Kissimmee, Florida, 34746
- Available
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
- Available
Nicklaus Children's Hospital
Miami, Florida, 33155
- Available
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Available
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
- Available
Gillette Children's
Saint Paul, Minnesota, 55101
- Available
Lucille Packard Children's Hospital at Stanford
San Carlos, California, 94070
- Available
UC San Diego Health
San Diego, California, 92121
- Available
Washington University
St Louis, Missouri, 63110
- Available
UMass Memorial Health Care
Worcester, Massachusetts, 01583
About this study
Official title: 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.
- Conditions
- Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn
- Being tested
- delpacibart zotadirsen
- 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
- Medical subject headings
- Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
delpacibart zotadirsenDrug
Also called Del-zota, AOC 1044
Del-zota is administered as an IV infusion every 6 weeks. Doses are administered based on body weight.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Disorders, AtrophicStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Avidity Biosciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Clinigen Healthcare Limited
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07250737
- Sponsor's protocol ID
- AOC 1044-MAP
- Status last verified
- Aug 2026
- First submitted
- 18 Nov 2025
- Last updated
- 8 Oct 2026
- Results posted
- No