Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
NCT ID: NCT07008469
Last Updated: 2025-12-22
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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ENROLLING_BY_INVITATION
PHASE3
230 participants
INTERVENTIONAL
2025-07-25
2030-10-31
Brief Summary
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Detailed Description
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Participants will receive an intravenous infusion of del-desiran at the clinical study site every 8 weeks for a total of 7 doses per year. The final dose will occur at Year 4, Visit 7, followed by a final assessment 8 weeks after the last dose.
An additional subgroup of de novo participants will also be included in a Fixed-Dose PK cohort.
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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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Del-desiran
Del-desiran (AOC 1001) will be administered seven times per year for up to 4 years.
Del-desiran (AOC 1001)
Del-desiran will be administered by intravenous (IV) infusion.
Interventions
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Del-desiran (AOC 1001)
Del-desiran will be administered by intravenous (IV) infusion.
Eligibility Criteria
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Inclusion Criteria
* Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia.
Exclusion Criteria
* Unwilling to comply with contraceptive requirements.
* Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study.
FIXED-DOSE PK COHORT
* Breastfeeding, pregnancy, or intent to become pregnant during the study
* Unwilling 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
16 Years
ALL
No
Sponsors
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Avidity Biosciences, Inc.
INDUSTRY
Responsible Party
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Locations
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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
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
Aomori Hospital
Aomori, Aomori, Japan
Countries
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Other Identifiers
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AOC 1001-CS4
Identifier Type: -
Identifier Source: org_study_id