Clinical Trial

Global Study of Del-desiran for the Treatment of DM1

Study acronym: HARBOR
Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1
Protocol Amendment History 24 amendments
This ClinicalTrials.gov record has been amended 24 times since 2024-05-08; most recent amendment 2026-01-20.
Status change: Recruiting → Active, Not Recruiting 2025-07-28
Status change: Not Yet Recruiting → Recruiting 2024-06-11
Trial Details
NCT Number NCT06411288
Lead Sponsor Avidity Biosciences, Inc.
Conditions DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease +2 more
Enrollment 159 participants
Start Date 2024-05-30
Primary Completion 2026-08 (estimated)
Study Completion 2026-09 (estimated)
Updated on ClinicalTrials.gov 2026-01-22