- recruitingPHASE2
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
This study is looking at a new medicine called PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1). Researchers want to see how safe it is and how well people tolerate it compared to a dummy medicine (placebo). It's for adults aged 16 to 65.
Canada · New Zealand · United Kingdom - recruitingPHASE1, PHASE2
Study of ATX-01 in Participants With DM1
This study is testing a new medicine, ATX-01, for Myotonic Dystrophy Type 1 (DM1). We want to see if it's safe and how people tolerate it. Participants will receive either ATX-01 or a dummy medicine called a placebo, in single or multiple doses.
United States · Canada · France - recruiting
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
This study aims to better understand Myotonic Dystrophy Type 1 (DM1) by collecting health information from adults with DM1. It will help researchers find better ways to measure the disease and how it progresses, which is important for developing new treatments.
United States · Canada · Germany - active not recruitingPHASE3
Global Study of Del-desiran for the Treatment of DM1
This study is testing a new medicine called del-desiran for Myotonic Dystrophy Type 1 (DM1). It's comparing the new medicine to a dummy treatment (placebo) to see if it's safe and helps people with DM1. Participants will receive infusions over about a year.
United States · Canada · Denmark