Global Open-Label Extension Study of Del-desiran for the Treatment of DM1 (NCT07008469) | Clinical Trial Compass
By InvitationPhase 3
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
United States230 participantsStarted 2025-07-25
Plain-language 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
Who can participate
Age range16 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
What they're measuring
1
TEAEs
Timeframe: From enrollment to the end of treatment at 4 years