Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

Running, not enrolling · Phase 2

Conditions studied: DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44

In brief

AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.

Key facts

Study ID

NCT06244082

Run by

Avidity Biosciences, Inc.

People needed

39

Starts

2024-01-22

Expected to finish

2027-04-30

Last updated by the study team

2026-07-30

Who can join

Age: 7 and older, up to 27. Sex: male. Healthy volunteers: not accepted.

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• University of California, San Diego, Rady's Children's Hospital — La Jolla, California, United States
• UC Davis Medical Center — Sacramento, California, United States
• Lucille Packard Children's Hospital at Stanford — San Carlos, California, United States
• Rare Disease Research - Atlanta — Atlanta, Georgia, United States
• University of Massachusetts Medical School — Worcester, Massachusetts, United States
• Gillette Children's Specialty Healthcare — Saint Paul, Minnesota, United States
• Rare Disease Research — Hillsborough, North Carolina, United States
• Abigail Wexner Research Institute at Nationwide Children's Hospital — Columbus, Ohio, United States
• Neurology Rare Disease Center — Denton, Texas, United States

Full record on ClinicalTrials.gov

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