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

NCT07008469 | Enrolling By Invitation Phase 3 DMC FDA Drug

• 1 other identifier: AOC 1001-CS4
• Study Type: interventional
• Target: 230
• Locations: 7 countries
• Sites: 26

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

Conditions

DM1; Myotonic Dystrophy; Myotonia; Myotonic Dystrophy 1; Myotonic Dystrophy Type 1; Myotonic Disorders; Steinert Myotonic Dystrophy; Steinert Disease

Keywords

DM1; Myotonic Dystrophy; Myotonic Dystrophy 1; Myotonia; Myotonic Dystrophy Type 1 (DM1); Dystrophy Myotonic; Myotonic Disorders; Steinert Disease; Steinert; Myotonic Muscular Dystrophy; HARBOR-OLE; Avidity Biosciences; Avidity; AOC 1001; Del-desiran; Delpacibart etedesiran; HARBOR

Outcome Measures

Primary Outcomes (1)

• TEAEs

  Incidence of treatment emergent adverse events

  From enrollment to the end of treatment at 4 years

Study Arms (1)

Del-desiran

EXPERIMENTAL

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

Drug: Del-desiran (AOC 1001)

Interventions

Del-desiran (AOC 1001) DRUG

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

Del-desiran

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• FIXED-DOSE PK COHORT
• Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia.
• Breastfeeding, pregnancy, or intent to become pregnant during the study
• Unwilling to comply with contraceptive requirements
• Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
• Diabetes that is not adequately controlled
• History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded
• Body Mass Index \> 35 kg/m2 at Screening
• Recently treated with an investigational drug or biological agent

Sponsors & Collaborators

• Avidity Biosciences, Inc.: lead

Study Sites (26)

University of California, Los Angeles (UCLA)

Los Angeles, California, 90095, United States

Location

Stanford University

Stanford, California, 94305, United States

Location

University of Colorado

Denver, Colorado, 80045, United States

Location

University of Florida

Gainesville, Florida, 32608, United States

Location

University Research Center of South Florida

Tampa, Florida, 33612, United States

Location

Indiana University (IU)

Indianapolis, Indiana, 46202, United States

Location

Kansas University Medical Center

Kansas City, Kansas, 66205, United States

Location

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Duke University Medical Center

Durham, North Carolina, 27708, United States

Location

Wake Forest

Winston-Salem, North Carolina, 27157, United States

Location

Ohio State University

Columbus, Ohio, 43221, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Houston Methodist Neurological Institute

Houston, Texas, 77030, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

University of Washington

Seattle, Washington, 98104, United States

Location

The Ottawa Hospital

Ottawa, Ontario, K1Y 4E9, Canada

Location

Montreal Neurological Institute

Montreal, Quebec, H3A 2B4, Canada

Location

AP-HP Hopital Pitie-Salpetriere

Paris, 75013, France

Location

Klinikum der Ludwig-Maximilians-Universitaet Muenchen

München, 80336, Germany

Location

Fondazione Serena Onlus - Centro Clinico NeMO Milano

Milan, 20162, Italy

Location

Aomori Hospital

Aomori, Aomori, 038-1331, Japan

Location

National Hospital Organization Osaka Toneyama Medical Center

Osaka, 560-8552, Japan

Location

Osaka University Hospital

Osaka, 565-0871, Japan

Location

Stichting Radboud Universitair Medisch Centrum

Nijmegen, 6525 GA, Netherlands

Location

MeSH Terms

Conditions

Myotonic Dystrophy; Myotonia; Myotonic Disorders

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neuromuscular Manifestations; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 29, 2025
• First Posted: June 6, 2025
• Study Start: July 25, 2025
• Primary Completion (Estimated): August 1, 2030
• Study Completion (Estimated): October 1, 2030
• Last Updated: April 23, 2026

Record last verified: 2026-04

Locations

US (17); JP (3); CA (2); NL (1); IT (1); FR (1); DE (1)