A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
Status: Active_not_recruiting
Location: See all (34) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1
Eligibility
Participation Requirements
Sex: All
Minimum Age: 16
Maximum Age: 65
Healthy Volunteers: f
View:
• Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1
• Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening
Locations
United States
California
Stanford University
Stanford
Colorado
University of Colorado
Denver
Florida
University of Florida
Gainesville
University Research Center of South Florida
Tampa
Indiana
Indiana University (IU)
Indianapolis
Kansas
Kansas University Medical Center
Kansas City
Massachusetts
Beth Israel Deaconess Medical Center
Boston
Maryland
Kennedy Krieger Institute
Baltimore
Minnesota
University of Minnesota
Minneapolis
North Carolina
Duke University Medical Center
Durham
Wake Forest
Winston-salem
New York
University of Rochester Medical Center
Rochester
Ohio
University of Cincinnati Gardner Neuroscience Institute
Cincinnati
Ohio State University
Columbus
Pennsylvania
University of Pennsylvania
Philadelphia
Texas
Houston Methodist Neurological Institute
Houston
Virginia
Virginia Commonwealth University
Richmond
Washington
University of Washington
Seattle
Other Locations
Canada
Montreal Neurological Institute
Montreal
The Ottawa Hospital
Ottawa
Denmark
Aarhus University Hospital
Aarhus N
Rigshospitalet
Copenhagen
France
AP-HP Hopital Pitie-Salpetriere
Paris
Germany
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
Munich
Italy
Fondazione Serena Onlus - Centro Clinico NeMO Milano
Milan
Japan
Aomori Hospital
Aomori
National Hospital Organization Osaka Toneyama Medical Center
Osaka
Osaka University Hospital
Osaka
National Center of Neurology and Psychiatry
Tokyo
Netherlands
Maastricht University Medical Center
Maastricht
Stichting Radboud Universitair Medisch Centrum
Nijmegen
Spain
Hospital Universitario Donostia
Donostia / San Sebastian
United Kingdom
St. Georges University Hospitals NHS Foundation Trust
London
University College London Hospital
London
Time Frame
Start Date: 2024-05-30
Completion Date: 2026-09
Participants
Target number of participants: 159
Treatments
Experimental: Del-desiran
Del-desiran (AOC 1001) will be administered seven times
Placebo_comparator: Placebo
Saline will be administered seven times
Related Therapeutic Areas
Sponsors
Leads: Avidity Biosciences, Inc.