This study involves people with Type 1 Myotonic Dystrophy, a genetic condition that causes muscle weakness and stiffness, particularly difficulty relaxing muscles after use. The study will test a new medicine called ARO-DM1, which is given through an infusion into a vein. Some participants will receive a placebo instead of the actual medicine. The medicine is being developed to target the genetic cause of this condition.
The purpose of this study is to find out if ARO-DM1 is safe and well-tolerated in people with Type 1 Myotonic Dystrophy, and to understand how the body processes the medicine and how it affects the disease. The study will also look at whether the medicine can improve the condition in some participants. This is a dose-escalating study, which means that different groups of participants will receive different amounts of the medicine to find the best dose. The study will measure how often side effects occur and how severe they are, as well as how the medicine moves through the bloodstream over time.
Participants in this study will be adults between 18 and 65 years old who have a confirmed genetic diagnosis of the condition with a specific genetic pattern. They must have visible signs of muscle stiffness and be able to walk independently for at least 10 meters, though they may use braces. The study involves receiving either the study medicine or placebo through infusions, and participants will be monitored throughout the study period for safety and how well the treatment works.
Belgium 
Germany 
Italy 
Spain 
