Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
Enrolling by invitation · Phase 3
Conditions studied: Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert Disease
In brief
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
Key facts
- Study ID
- NCT07008469
- Run by
- Avidity Biosciences, Inc.
- People needed
- 230
- Starts
- 2025-06-26
- Expected to finish
- 2030-10-01
- Last updated by the study team
- 2026-07-23
Who can join
Age: 16 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- 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
- University Research Center of South Florida — Tampa, 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 Minnesota — Minneapolis, Minnesota, United States
- University of Rochester Medical Center — Rochester, New York, United States
- Duke University Medical Center — Durham, North Carolina, United States
- Wake Forest — Winston-Salem, North Carolina, United States
- University of Cincinnati Gardner Neuroscience Institute — Cincinnati, Ohio, 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
- University of Washington — Seattle, Washington, United States
- The Ottawa Hospital — Ottawa, Ontario, Canada
- Montreal Neurological Institute — Montreal, Quebec, Canada
- Aarhus University Hospital — Aarhus, Denmark
- Rigshospitalet — Copenhagen, Denmark
- AP-HP Hopital Pitie-Salpetriere — Paris, France
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen — München, Germany
Full record on ClinicalTrials.gov
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