drugset
drugset / Trial / NCT05479981

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

NCT05479981 ↗

Phase 2 Completed 37 enrolled Avidity Biosciences, Inc.
Non-randomizedParallel-groupOpen-labelTreatment

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

Timeline

Start
2022-08-04
Primary completion
2025-09-05
Completion
2025-09-10

Drugs

EvaluationDrugModalityDoseRoute
Subject AOC 1001 Unknown — Intravenous

Indications

atrophic muscular disease inherited neurodegenerative disorder myotonic dystrophy type 1