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

NCT ID NCT06244082

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

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.

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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&#39;s Hospital

Little Rock, Arkansas, 72202, United States
Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, 55101, United States
Lucille Packard Children&#39;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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