A Phase 2 interventional study of Placebo and AOC 1001 in DM1, Muscular Dystrophies and Myotonic Dystrophy, sponsored by Avidity Biosciences, Inc.. Completed at 8 sites in United States. Open to participants aged 18 Years to 66 Years. Per ClinicalTrials.gov, last updated 2026-09-11.
Sponsored by Avidity Biosciences, Inc. · Phase 2, Interventional, and Treatment
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) 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
This study will continue to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AOC 1001 in participants that enrolled in the randomized, placebo-controlled, First-In-Human Phase 1/2 AOC 1001-CS1 (MARINA) (NCT05027269) clinical study.
Participants from AOC 1001-CS1 (NCT05027269) are eligible to enroll in AOC 1001-CS2 (NCT05479981) if they have satisfactorily completed AOC 1001-CS1 (NCT05027269).
After completion of Q8W (once every 8 weeks) Visit 6 assessments, participants will have the option to enroll in another AOC 1001 open-label extension (OLE) study - AOC 1001-CS4 (HARBOR-OLE) (NCT07008469). 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.
Key Inclusion Criteria:
Key Exclusion Criteria:
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.
Drug: 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 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.
Drug: Placebo · Drug: AOC 1001
Placebo will be administered by intravenous (IV) infusion.
Also known as: Saline
AOC 1001 will be administered by intravenous (IV) infusion.
Number and severity of treatment-emergent adverse events (TEAEs)
Time frame: Through study completion
AOC 1001 levels in muscle tissue
Time frame: Through Day 183
Change and percentage change from baseline in DMPK mRNA knockdown
Time frame: Through Day 183
Change and percentage change from baseline in Spliceopathy
Time frame: Through Day 183
Plasma pharmacokinetic (PK) parameters
Maximum and trough plasma concentration
Time frame: Up to Day 729
This study is completed, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Avidity Biosciences, Inc.