Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
159 participants
INTERVENTIONAL
2024-05-30
2026-09-30
Brief Summary
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Detailed Description
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Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at the clinical study site every 8 weeks for a total of 7 doses. The final dose will occur at Week 48, followed by a final assessment at Week 54.
After completion of Week 54 assessments, eligible participants will have the option to enroll into an open label extension (OLE) study, pending regulatory approval.
An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Del-desiran
Del-desiran (AOC 1001) will be administered seven times
AOC 1001 (del-desiran)
Del-desiran will be administered by intravenous (IV) infusion.
Placebo
Saline will be administered seven times
Placebo
Placebo will be administered by intravenous (IV) infusion.
Interventions
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AOC 1001 (del-desiran)
Del-desiran will be administered by intravenous (IV) infusion.
Placebo
Placebo will be administered by intravenous (IV) infusion.
Eligibility Criteria
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Inclusion Criteria
* Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening
Exclusion Criteria
* Unwilling or unable to comply with contraceptive requirements
* Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
* Diabetes that is not adequately controlled
* History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded.
* Body Mass Index \> 35 kg/m2 at Screening
* Recently treated with an investigational drug or biological agent
* Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.
16 Years
65 Years
ALL
No
Sponsors
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Avidity Biosciences, Inc.
INDUSTRY
Responsible Party
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Locations
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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 N, , Denmark
Rigshospitalet
Copenhagen, , Denmark
AP-HP Hopital Pitie-Salpetriere
Paris, , France
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
Munich, , Germany
Fondazione Serena Onlus - Centro Clinico NeMO Milano
Milan, , Italy
Aomori Hospital
Aomori, Aomori, Japan
National Hospital Organization Osaka Toneyama Medical Center
Osaka, , Japan
Osaka University Hospital
Osaka, , Japan
National Center of Neurology and Psychiatry
Tokyo, , Japan
Maastricht University Medical Center
Maastricht, , Netherlands
Stichting Radboud Universitair Medisch Centrum
Nijmegen, , Netherlands
Hospital Universitario Donostia
Donostia / San Sebastian, , Spain
University College London Hospital
London, , United Kingdom
St. Georges University Hospitals NHS Foundation Trust
London, , United Kingdom
Countries
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Other Identifiers
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AOC 1001-CS3
Identifier Type: -
Identifier Source: org_study_id
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