Global Study of Del-desiran for the Treatment of DM1 (NCT06411288) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Global Study of Del-desiran for the Treatment of DM1
United States159 participantsStarted 2024-05-30
Plain-language summary
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
Who can participate
Age range16 Years – 65 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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