This study is testing an investigational drug called zeleciment basivarsen (DYNE-101) for people with Myotonic Dystrophy Type 1 (DM1), also known as Steinert Disease. Researchers want to see how well it works, if it's safe, and if people can tolerate it. You might be able to join if you have a confirmed diagnosis of DM1 with a specific genetic marker (trinucleotide repeat size greater than 100), are able to walk 10 meters and complete a 5 times sit-to-stand test independently, and have a body mass index (BMI) less than 35. The main way they will measure if the treatment is working is by timing how long it takes you to stand up from a chair and sit down five times (5 Times Sit-To-Stand, or 5xSTS) at the beginning and after 49 weeks. The study is currently recruiting about 150 participants.

Study design

This interventional study involves giving participants either zeleciment basivarsen (DYNE-101) or a placebo (an inactive substance) through an IV infusion. It includes a placebo-controlled period and a long-term extension period.

What's involved

The study involves a screening period of up to 8 weeks, followed by a 48-week placebo-controlled period, and then a 24-week long-term extension period.

Compensation

Not stated in the trial record.

Follow-up

The primary outcome measure (5 Times Sit-To-Stand Time) is measured at Baseline and Week 49.

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