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Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Sponsor: Avidity Biosciences, Inc.
Summary
AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
Official title: A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
Key Details
Gender
All
Age Range
18 Years - 66 Years
Study Type
INTERVENTIONAL
Enrollment
37
Start Date
2022-08-04
Completion Date
2025-09-10
Last Updated
2026-09-08
Healthy Volunteers
No
Conditions
Interventions
Placebo
Placebo will be administered by intravenous (IV) infusion.
AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
Locations (8)
University of California Los Angeles
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
University of Colorado
Denver, Colorado, United States
University of Florida
Gainesville, Florida, United States
Kansas University Medical Center
Kansas City, Kansas, United States
University of Rochester Medical Center
Rochester, New York, United States
Ohio State University
Columbus, Ohio, United States
Virginia Commonwealth University
Richmond, Virginia, United States