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Clinical Trials in the USA / NCT07008469
Enrolling by invitation Phase 3

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

NCT07008469 · tracked via the Priya Life Science USA tracker
Sponsor
Avidity Biosciences, Inc.
Phase
Phase 3
Started
2025-06-26
Last updated
2026-08-19

Condition(s) studied

Myotonic Dystrophy Type 1DM1Myotonic DystrophyMyotoniaMyotonic Dystrophy 1Myotonic DisordersSteinert Myotonic DystrophySteinert Disease

Investigational drug(s) / intervention(s)

Del-desiran (AOC 1001)

Del-desiran (AOC 1001): Del-desiran will be administered by intravenous (IV) infusion.

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

Eligibility

Sex
ALL
Min age
16 Years
Max age
Healthy volunteers
No
FROM A PARENT STUDY Key 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. FIXED-DOSE PK COHORT Key Inclusion Criteria: * Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia. Key Exclusion Criteria: * 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 Note: Additional protocol defined Inclusion and Exclusion criteria apply

Primary outcome measure(s)

Trial sites (35)

FacilityCityRegionStatus
University of California, Los Angeles (UCLA) Los Angeles California
Stanford University Stanford California
University of Colorado Denver Colorado
University of Florida Gainesville Florida
University Research Center of South Florida Tampa Florida
Indiana University (IU) Indianapolis Indiana
Kansas University Medical Center Kansas City Kansas
Kennedy Krieger Institute Baltimore Maryland
Beth Israel Deaconess Medical Center Boston Massachusetts
University of Minnesota Minneapolis Minnesota
University of Rochester Medical Center Rochester New York
Duke University Medical Center Durham North Carolina
Wake Forest Winston-Salem North Carolina
University of Cincinnati Gardner Neuroscience Institute Cincinnati Ohio
Ohio State University Columbus Ohio
University of Pennsylvania Philadelphia Pennsylvania
Houston Methodist Neurological Institute Houston Texas
Virginia Commonwealth University Richmond Virginia
University of Washington Seattle Washington
The Ottawa Hospital Ottawa Ontario
Montreal Neurological Institute Montreal Quebec
Aarhus University Hospital Aarhus Denmark
Rigshospitalet Copenhagen Denmark
AP-HP Hopital Pitie-Salpetriere Paris France
Klinikum der Ludwig-Maximilians-Universitaet Muenchen München Germany
Fondazione Serena Onlus - Centro Clinico NeMO Milano Milan Italy
Aomori Hospital Aomori Aomori
National Hospital Organization Osaka Toneyama Medical Center Osaka Japan
Osaka University Hospital Osaka Japan
National Center of Neurology and Psychiatry Tokyo Japan
Maastricht University Medical Center Maastricht Netherlands
Stichting Radboud Universitair Medisch Centrum Nijmegen Netherlands
Hospital Universitario Donostia San Sebastián Spain
University College London Hospital London United Kingdom
St. Georges University Hospitals NHS Foundation Trust London United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07008469 on ClinicalTrials.gov ↗ ← All trials in the USA