Myotonic Dystrophy Type 1 research hub
4 indexed studies · 3 currently recruiting
- recruitingTherapeutic confirmatory (Phase III)
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
This phase 3 clinical trial is testing a new medicine called AOC 1001 for adults with myotonic dystrophy type 1. Researchers want to see if the medicine, given through a vein drip, helps improve muscle stiffness in the hands, increases strength, and improves daily life over 54 weeks.
Netherlands · France · Spain - recruitingNA
Pelvic Floor Muscle Training for Women with Myotonic Dystrophy
This study looks at how pelvic floor muscle training can help women with Myotonic Dystrophy Type 1 who experience bladder leaks (urinary incontinence). It aims to see if these exercises are helpful, easy to do, and can improve their quality of life.
Canada - enrolling by invitationPHASE3
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
This study is an extension of previous research looking at a new medicine called del-desiran for Myotonic Dystrophy Type 1 (DM1). It aims to check its long-term safety and how well it works over several years.
United States · Canada · Denmark - authorisedTherapeutic confirmatory (Phase III)
A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1
This study is testing a new medicine, DYNE-101, for people with Myotonic Dystrophy Type 1. It compares the new medicine to a dummy treatment over 48 weeks to see if it's safe and helps improve daily activities.
Netherlands · Spain · Italy