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Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
Sponsor: Avidity Biosciences, Inc.
Summary
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.
Official title: A Phase 2 Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants With Mutations Amenable to Exon 44 Skipping
Key Details
Gender
MALE
Age Range
7 Years - 27 Years
Study Type
INTERVENTIONAL
Enrollment
39
Start Date
2024-01-22
Completion Date
2027-07-31
Last Updated
2025-05-14
Healthy Volunteers
No
Interventions
AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion
Locations (10)
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