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Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
Sponsor: Avidity Biosciences, Inc.
Summary
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1
Official title: A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
Key Details
Gender
All
Age Range
16 Years - Any
Study Type
INTERVENTIONAL
Enrollment
230
Start Date
2025-07-25
Completion Date
2030-10
Last Updated
2026-02-03
Healthy Volunteers
No
Conditions
Interventions
Del-desiran (AOC 1001)
Del-desiran will be administered by intravenous (IV) infusion.
Locations (20)
University of California, Los Angeles (UCLA)
Los Angeles, California, United States
Stanford University
Stanford, California, United States
University of Colorado
Denver, Colorado, United States
University of Florida
Gainesville, Florida, United States
Indiana University (IU)
Indianapolis, Indiana, United States
Kansas University Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States
University of Rochester Medical Center
Rochester, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Ohio State University
Columbus, Ohio, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Houston Methodist Neurological Institute
Houston, Texas, United States
Virginia Commonwealth University
Richmond, Virginia, United States
The Ottawa Hospital
Ottawa, Ontario, Canada
Montreal Neurological Institute
Montreal, Quebec, Canada
Fondazione Serena Onlus - Centro Clinico NeMO Milano
Milan, Italy
Aomori Hospital
Aomori, Aomori, Japan
Osaka University Hospital
Osaka, Japan
Stichting Radboud Universitair Medisch Centrum
Nijmegen, Netherlands