Global Study of Del-desiran for the Treatment of DM1
Myotonic Dystrophy 1 | Steinert Disease | Myotonic Dystrophy | DM1 | Myotonic Muscular Dystrophy | Myotonic...A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1
null
Participation Requirements
-
Sex:
ALL -
Eligible Ages:
16 to 65
Participation Criteria
Key Inclusion Criteria:
* Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1
* Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening
Key Exclusion Criteria:
* Breastfeeding, pregnancy, or intent to become pregnant during the study
* Unwilling or unable 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
* Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.
Note: Additional protocol defined Inclusion and Exclusion criteria apply
Study Location
The Ottawa Hospital
The Ottawa HospitalOttawa, Ontario
Canada
Contact Study Team
CSSS de Chicoutimi
CSSS de ChicoutimiChicoutimi, Quebec
Canada
Contact Study Team
Montreal Neurological Institute
Montreal Neurological InstituteMontreal, Quebec
Canada
Contact Study Team
- Study Sponsored By
- Avidity Biosciences, Inc.
- Participants Required
- More Information
- Study ID:
NCT06411288