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. (part of Novartis) · 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.
126 studies on the registry are indexed under Myotonic Dystrophy; 50 are open to participants now.
This study's enrollment of 37 is close to the median of 40 across 67 interventional studies indexed under Myotonic Dystrophy.
Browse Myotonic Dystrophy studies →Avidity Biosciences, Inc. is the lead sponsor of 11 studies on the registry; 4 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.
First submitted before Avidity Biosciences, Inc. became part of Novartis.
Compared within Myotonic Dystrophy, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Muscular Dystrophies. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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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Studies of the drug tested here: AOC 1001