NCT05027269

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

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 39 (actual)
• Sponsor: Avidity Biosciences, Inc. · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• DM1
• Myotonic Dystrophy 1
• Myotonic Dystrophy
• Myotonic Dystrophy Type 1 (DM1)
• Dystrophy Myotonic
• Myotonic Disorders
• Steinert Disease
• Myotonic Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	AOC 1001	AOC 1001 will be administered by intravenous (IV) infusion.
DRUG	Placebo	Placebo will be administered by intravenous (IV) infusion.

Timeline

• Start date: 2021-10-28
• Primary completion: 2023-02-14
• Completion: 2023-02-14
• First posted: 2021-08-30
• Last updated: 2026-03-12

Locations

8 sites across 1 country: United States

Regulatory

• FDA-regulated drug study