This study is testing a drug called ATX-01 in people with Myotonic Dystrophy Type 1 (DM1). DM1 is a genetic condition that causes muscle weakness and other problems. The main goal is to see if ATX-01 is safe and well-tolerated. Researchers will compare ATX-01 to a placebo (an inactive substance) to understand its effects. ATX-01 works by blocking a specific microRNA called miR-23b. To join, you must be between 18 and 64 years old, have a confirmed DM1 diagnosis with a specific genetic marker (CTG expansion of >150 repeats in the DMPK gene), and be able to walk 10 meters without assistance (except for ankle-foot orthoses). The study is currently unclear about its recruitment status and plans to enroll 56 participants.

Study design

This is an interventional study comparing ATX-01 to a placebo. It will involve a single-ascending dose part and a multiple-ascending dose part, with 56 planned participants.

What's involved

In the single-ascending dose part, participants will receive one dose of ATX-01 or placebo. In the multiple-ascending dose part, participants will receive three doses of ATX-01 or placebo.

Compensation

Not stated in the trial record.

Follow-up

Participants will be monitored for adverse events for up to 120 days.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.