Clinical Trial

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

Study acronym: HARBOR-OLE
Enrolling by Invitation Phase 3
View on ClinicalTrials.gov →
Summary
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1
Protocol Amendment History 2 changes
notable Trial sites expanded: 26 -> 35 locations 2026-07-24
notable Trial sites expanded: 20 -> 26 locations 2026-04-24
Trial Details
NCT Number NCT07008469
Lead Sponsor Avidity Biosciences, Inc.
Conditions Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert Disease
Enrollment 230 participants
Start Date 2025-06-26
Primary Completion 2030-08 (estimated)
Study Completion 2030-10 (estimated)
Updated on ClinicalTrials.gov 2026-07-23