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Can a targeted infusion slow muscle decline in duchenne? a new trial aims to find out.

NCT ID NCT06244082

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 This study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 Jul 30, 2026 · Last updated Jul 31, 2026 · Updated 1 time

Summary

This phase 2 trial tests an experimental drug called AOC 1044 in people with Duchenne muscular dystrophy whose genetic mutation makes them candidates for exon 44 skipping therapy. The drug is given by intravenous infusion. Researchers are evaluating long-term safety and whether it can reduce levels of creatine kinase, a marker of muscle damage. Participants include both those who previously received AOC 1044 and new volunteers aged 7 to 27.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
an experimental drug called AOC 1044, given by intravenous infusion
What this could lead to
If it works, this could point toward a treatment that slows muscle damage in people with Duchenne muscular dystrophy who have a specific genetic mutation.
What could go wrong
This is an early-phase, open-label trial with no placebo group, so results may be hard to interpret. Long-term safety and effectiveness are not yet known.

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.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

39 people

The number who actually took part.

Started

Jan 2024

Expected to finish

Apr 2027

An estimate. End dates often move.

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

7 to 27 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

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: * Satisfactorily completed AOC 1044-CS1 (EXPLORE44) as determined by the Investigator and Sponsor * No significant tolerability issues with AOC 1044 De novo Participants: * Aged 7 to 27 years, inclusive, at the time of informed consent * Clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years * Confirmation of DMD gene mutation amenable to exon 44 skipping * Weight ≥ 23 kg * Ambulatory or non-ambulatory * Ambulatory participants: LVEF ≥50% and FVC≥50% * Non-ambulatory participants: LVEF ≥45% and FVC≥40% * PUL 2.0 entry item A ≥3 * If on corticosteroids, stable dose for 30 days before screening and throughout the study Key Exclusion Criteria Rollover Participants: * Presence of any new condition or worsening of existing condition that could affect a participant\'s safety or ability to comply with study procedures De novo Participants: * Serum hemoglobin \< lower limit of normal * Uncontrolled hypertension or diabetes * Prior treatment with any cell or gene therapy * Prior treatment with another exon 44 skipping agent within 6 months prior to informed consent * Recently treated with an investigational drug * History of multiple drug allergies

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

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

DMD Duchenne Duchenne muscular dystrophy Exon 44

Contacts and locations

Locations

  • Abigail Wexner Research Institute at Nationwide Children's Hospital

    Columbus, Ohio, 43215, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Gillette Children's Specialty Healthcare

    Saint Paul, Minnesota, 55101, United States

  • Lucille Packard Children's Hospital at Stanford

    San Carlos, California, 94070, United States

  • Neurology Rare Disease Center

    Denton, Texas, 76208, United States

  • Rare Disease Research

    Hillsborough, North Carolina, 27278, United States

  • Rare Disease Research - Atlanta

    Atlanta, Georgia, 30329, United States

  • UC Davis Medical Center

    Sacramento, California, 95817, United States

  • University of California, San Diego, Rady's Children's Hospital

    La Jolla, California, 92037, United States

  • University of Massachusetts Medical School

    Worcester, Massachusetts, 01655, United States

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