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NCT07008469Phase 3ENROLLING BY INVITATION

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

Study 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

Conditions
Myotonic Dystrophy Type 1DM1Myotonic DystrophyMyotoniaMyotonic Dystrophy 1Myotonic DisordersSteinert Myotonic DystrophySteinert Disease
Interventions / Drugs
Del-desiran (AOC 1001)
View on ClinicalTrials.gov ↗
Sponsor
RNA
Avidity Biosciences
RNA Therapeutics
C51/100
Study Details
Enrollment230 participants
Primary Completion—
Lead SponsorAvidity Biosciences, Inc.
Data Retrieved2026-04-11 04:26:08.607659+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Current status: ENROLLING BY INVITATION.

Enrolled: 230 participants
Sponsor Catalyst Profile

Avidity Biosciences (RNA) is the sponsoring company for this trial. BiotechSign currently grades this company C (51/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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