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