Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Completed · Phase 1/Phase 2 · Has a placebo group

Conditions studied: DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Myotonic Muscular Dystrophy

In brief

AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA). Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period. Part B is a multiple-ascending dose design with 2 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.

Key facts

Study ID

NCT05027269

Run by

Avidity Biosciences, Inc.

People needed

39

Starts

2021-10-28

Expected to finish

2023-02-14

Last updated by the study team

2026-03-12

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

Where it is running

• 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

Full record on ClinicalTrials.gov

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