Myotonic Dystrophy Type 1 (DM1) research hub
6 indexed studies · 4 currently recruiting
- recruitingPHASE3
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
This study looks at a new medicine called zeleciment basivarsen (DYNE-101) for Myotonic Dystrophy Type 1. We want to see if it's safe, works well, and how people tolerate it. Participants will receive either the study medicine or a dummy medicine for nearly a year.
United States · Belgium · Denmark - 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 - enrolling by invitationPHASE2
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
This study is looking at how safe and effective a new medicine called VX-670 is for people with Myotonic Dystrophy Type 1 over a long time. It only includes people who have already been in another study for this medicine.
Australia · Belgium · Canada - recruitingPHASE1, PHASE2
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
This study is looking at a new medicine called VX-670 for Myotonic Dystrophy Type 1 (DM1). Researchers want to see if it's safe, how the body handles it, and if it works for people aged 18-64. Some participants will receive the medicine, others a placebo.
United States · Australia · Belgium - recruitingPHASE1, PHASE2
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
This study is looking at a new medicine called DYNE-101 for people with Myotonic Dystrophy Type 1 (DM1). We want to check if it's safe, how well people tolerate it, and if it helps with the condition. It involves injections and visits over a long period.
United States · Australia · France - active not recruitingNA
A Remote Physical Activity Program in the Population Suffering from Type 1 Myotonic Dystrophy
This study looks at a new online exercise programme for people with Myotonic Dystrophy Type 1 (DM1). It aims to see if the 'PACE' tool can help them stay active and improve their health, especially after challenges like the COVID-19 pandemic.
Canada