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Cure My Disease

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New hope for duchenne: targeted therapy now available for eligible patients

NCT ID NCT07250737

Disease control Sponsor: Avidity Biosciences, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access This study
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 02, 2026 · Updated 10 times

Summary

This program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease and improve muscle function. Participants must be at least 2 years old and live in the US. This is not a cure, but aims to manage the condition long-term.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 years and older

Sex

Male participants only

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

atrophic muscular disease Duchenne muscular dystrophy hereditary disease muscle tissue disorder Muscular Disorders, Atrophic muscular dystrophy musculoskeletal system disorder nervous system disorder neuromuscular disease X-linked disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Genetic diseases, inborn Genetic diseases, X-linked Muscular diseases Muscular disorders, atrophic Muscular dystrophies Muscular dystrophy, Duchenne Musculoskeletal diseases Nervous system diseases Neuromuscular diseases

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    21 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Arkansas Children's Hospital

    AVAILABLE

    Little Rock, Arkansas, 72202, United States

  • Children's Hospital of Philadelphia

    AVAILABLE

    Philadelphia, Pennsylvania, 19104, United States

  • Connecticut Children's Medical Center

    AVAILABLE

    Hartford, Connecticut, 06106, United States

  • Gillette Children's

    AVAILABLE

    Saint Paul, Minnesota, 55101, United States

  • Lucille Packard Children's Hospital at Stanford

    AVAILABLE

    San Carlos, California, 94070, United States

  • Nationwide Children's Hospital

    AVAILABLE

    Columbus, Ohio, 43205, United States

  • Neurology & Neuromuscular Care Center

    AVAILABLE

    Flower Mound, Texas, 75028, United States

  • Nicklaus Children's Hospital

    AVAILABLE

    Miami, Florida, 33155, United States

  • Prisma Health-Midlands Children's Hospital

    AVAILABLE

    Columbia, South Carolina, 29203, United States

  • Rare Disease Research

    AVAILABLE

    Kissimmee, Florida, 34746, United States

  • Rare Disease Research

    AVAILABLE

    Atlanta, Georgia, 30329, United States

  • Rare Disease Research

    AVAILABLE

    Iselin, New Jersey, 08830, United States

  • Rare Disease Research

    AVAILABLE

    Hillsborough, North Carolina, 27278, United States

  • UC San Diego Health

    AVAILABLE

    San Diego, California, 92121, United States

  • UCI Alpha Clinic

    AVAILABLE

    Irvine, California, 92612, United States

  • UMass Memorial Health Care

    AVAILABLE

    Worcester, Massachusetts, 01583, United States

  • University of Alabama

    AVAILABLE

    Birmingham, Alabama, 35294, United States

  • University of Kansas Medical Center

    AVAILABLE

    Kansas City, Kansas, 66103, United States

  • University of Pennsylvania

    AVAILABLE

    Philadelphia, Pennsylvania, 19104, United States

  • University of Vermont Medical Center

    AVAILABLE

    Burlington, Vermont, 05401, United States

  • Washington University

    AVAILABLE

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.