AOC 1001-CS3

About this trial

Last updated 6 months ago

Study ID

AOC 1001-CS3

Status

Recruiting

Type

Interventional

Phase

Phase 3

Placebo

Yes

Accepting

16 to 65 Years

All

Not accepting

Healthy Volunteers

Trial Timing

Started a year ago

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

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

Global Study of Del-desiran for the Treatment of DM1