Global Study of Del-desiran for the Treatment of DM1

About

Brief 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

Primary Purpose

Treatment

Study Type

Interventional

Phase

Phase 3

Eligibility

Gender

All

Healthy Volunteers

No

Minimum Age

16 Years

Maximum Age

65 Years

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.