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ENROLLING BY INVITATION
NCT07008469
PHASE3

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

Sponsor: Avidity Biosciences, Inc.

View on ClinicalTrials.gov

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

Interventions

DRUG

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