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🔬NCT06411288Phase 3ACTIVE NOT RECRUITING

Global Study of Del-desiran for the Treatment of DM1

Study 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

Conditions
DM1Myotonic DystrophyMyotonic Dystrophy 1MyotoniaMyotonic Dystrophy Type 1 (DM1)Dystrophy MyotonicMyotonic DisordersSteinert DiseaseSteinertMyotonic Muscular Dystrophy
Interventions / Drugs
AOC 1001 (del-desiran)Placebo
View on ClinicalTrials.gov ↗
Sponsor
RNA
Avidity Biosciences
RNA Therapeutics
D48/100
Study Details
Enrollment159 participants
Primary Completion—
Lead SponsorAvidity Biosciences, Inc.
Data Retrieved2026-04-11 04:26:08.607643+00:00
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