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

Phase 3

• Conditions: Myotonic Dystrophy Type 1; DM1; Myotonia; Myotonic Dystrophy 1; Myotonic Dystrophy; Myotonic Disorders; Steinert Myotonic Dystrophy; Steinert's Disease
• Interventions: Drug: Del-desiran (AOC 1001)

• Registration Number: NCT07008469
• Lead Sponsor: Avidity Biosciences, Inc.

Brief 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

Detailed Description

The study consists of a Screening Period of up to either 4-weeks or 8-weeks depending on the prior parent trial, and up to a 4-year Treatment Period. The anticipated duration is 50 months and 2 weeks (4 years and 2.5 months).

Participants will receive an intravenous infusion of del-desiran at the clinical study site every 8 weeks for a total of 7 doses per year. The final dose will occur at Year 4, Visit 7, followed by a final assessment 8 weeks after the last dose.

An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.

Study Timeline

• Study First Submitted: 2025-05-29
• Study First Submitted QC: 2025-05-29
• Study First Posted: 2025-06-06
• Study Start: 2025-07-25
• Status Verified: 2025-08
• Last Update Submitted: 2025-08-29
• Last Update Posted: 2025-09-05
• Primary Completion: 2030-08
• Study Completion: 2030-10-01

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 217

Inclusion Criteria

• Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator.

Key

Exclusion Criteria

• Breastfeeding, pregnancy, or intent to become pregnant during the study.
• Unwilling to comply with contraceptive requirements.
• Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Del-desiran	Del-desiran (AOC 1001)	Del-desiran (AOC 1001) will be administered seven times per year for up to 4 years.

Primary Outcome Measures

Name	Time	Method
TEAEs	From enrollment to the end of treatment at 4 years	Incidence of treatment emergent adverse events

Trial Locations

Locations (9)

University of California, Los Angeles (UCLA); 🇺🇸 Los Angeles, California, United States

Stanford University; 🇺🇸 Stanford, California, United States

University of Colorado; 🇺🇸 Denver, Colorado, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

Kansas University Medical Center; 🇺🇸 Kansas City, Kansas, United States

University of Rochester Medical Center; 🇺🇸 Rochester, New York, United States

Ohio State University; 🇺🇸 Columbus, Ohio, United States

Houston Methodist Neurological Institute; 🇺🇸 Houston, Texas, United States

Virginia Commonwealth University; 🇺🇸 Richmond, Virginia, United States