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

About

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

Primary Purpose
Treatment
Study Type
Interventional
Phase
Phase 3

Eligibility

Gender
All
Healthy Volunteers
No
Minimum Age
16 Years
Maximum Age
N/A

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

Join this Trial

Enrolling by Invitation
These studies are not open to everyone who meets the eligibility criteria, but only to people in that particular population, who are specifically invited to participate.
Study Stats
Protocol No.
25-0779
Category
Brain/Neurological Diseases
Genetic and Rare Diseases
Musculoskeletal Disorders
Principal Investigator
Contact
Kaitlyn Kirchhoffer
Location
  • UCLA Westwood
For Providers
NCT No.
NCT07008469
For detailed technical eligibility, visit ClinicalTrials.gov.