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
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
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.
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
Location
- UCLA Westwood