Join us at Health Research Day — June 6th at Canton Waterfront Park, Baltimore!   Learn More →
← Back to all trials
Enrolling By Invitation NCT07008469

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

Conditions: Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert Disease

Sex: All
Ages: 16 Years – N/A
Healthy volunteers: No
Phase: PHASE3
Enrollment: 230
Sponsor: Avidity Biosciences, Inc.

Location: University of California, Los Angeles (UCLA) Los Angeles California

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 Criteria

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

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT07008469). StuddyBuddy aggregates publicly available trial information.