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Global Study of Del-desiran for the Treatment of DM1

NCT06411288 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 159

Last updated 2026-01-22

No results posted yet for this 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

  • DM1
  • Myotonic Dystrophy
  • Myotonic Dystrophy 1
  • Myotonia
  • Myotonic Dystrophy Type 1 (DM1)
  • Dystrophy Myotonic
  • Myotonic Disorders
  • Steinert Disease
  • Steinert
  • Myotonic Muscular Dystrophy

Interventions

DRUG

AOC 1001 (del-desiran)

Del-desiran will be administered by intravenous (IV) infusion.

DRUG

Placebo

Placebo will be administered by intravenous (IV) infusion.

Sponsors & Collaborators

  • Avidity Biosciences, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
16 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-05-30
Primary Completion
2026-08-31
Completion
2026-09-30
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Denmark
  • France
  • Germany
  • Italy
  • Japan
  • Netherlands
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06411288 on ClinicalTrials.gov