Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
NCT ID: NCT05479981
Last Updated: 2025-10-08
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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COMPLETED
PHASE2
37 participants
INTERVENTIONAL
2022-08-04
2025-09-10
Brief Summary
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Detailed Description
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Participants from AOC 1001-CS1 are eligible to enroll in AOC 1001-CS2 if they have satisfactorily completed AOC 1001-CS1.
After completion of Q8W Visit 6 assessments, participants will have the option to enroll in another AOC 1001 open-label extension (OLE) study. Participants who do not enroll in an AOC 1001 OLE study will be monitored for safety for a total of 8 weeks from the last dose of study drug. Total duration on study is up to 37 months (3 years 1 month).
As of September 2024, the dosing regimen was updated to every 8 weeks.
Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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AOC 1001
AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.
AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
AOC 1001 (with Placebo at Day 43)
AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.
Placebo
Placebo will be administered by intravenous (IV) infusion.
AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
Interventions
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Placebo
Placebo will be administered by intravenous (IV) infusion.
AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
Exclusion Criteria
* Unwilling or unable to continue to comply with contraceptive requirements
* Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study
18 Years
66 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
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
Countries
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Other Identifiers
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AOC 1001-CS2
Identifier Type: -
Identifier Source: org_study_id
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