NCT05479981

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 37 (actual)
• Sponsor: Avidity Biosciences, Inc. · Industry
• Sex: All
• Age: 18 Years – 66 Years
• Healthy volunteers: Not accepted

Summary

AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) 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

Detailed description

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) clinical study. 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

• DM1
• Muscular Dystrophies
• Myotonic Dystrophy
• Myotonic Dystrophy 1
• Myotonic Disorders
• Muscular Disorders, Atrophic
• Muscular Diseases
• Musculoskeletal Diseases
• Neuromuscular Diseases
• Nervous System Diseases
• Genetic Diseases, Inborn
• Heredodegenerative Disorders, Nervous System
• Neurodegenerative Diseases

Interventions

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

Timeline

• Start date: 2022-08-04
• Primary completion: 2025-09-05
• Completion: 2025-09-10
• First posted: 2022-07-29
• Last updated: 2025-10-08

Locations

8 sites across 1 country: United States

Regulatory

• FDA-regulated drug study