- active not recruiting
An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders
This study looks at how safe and effective a medicine called Namuscla (mexiletine) is for adults with non-dystrophic myotonic disorders. It's an observational study, meaning researchers will watch and collect information from patients already taking the medicine, or who start taking it, in their normal care.
France · Germany · United Kingdom - recruiting
The United Kingdom National Registry for Myotonic Dystrophy
This UK registry collects information from people with Myotonic Dystrophy (DM1 or DM2). It helps researchers understand the condition better and find people who might be interested in future studies or clinical trials to develop new treatments.
United Kingdom - 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 - recruitingPHASE3
The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
This study is testing a new form of mexiletine, a medicine, for people with Myotonic Dystrophy Types 1 and 2. It will compare the new medicine to a dummy pill (placebo) to see if it helps with muscle problems and if it's safe. It lasts for 26 weeks.
Belgium · Denmark · Germany - recruitingPHASE1, PHASE2
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
This study is testing a new gene therapy called SAR446268 for myotonic dystrophy type 1. It aims to see if it's safe, and if it can improve muscle function in people aged 10-55 with the condition.
United States · Argentina · Australia - 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 - enrolling by invitationPHASE3
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study.
This study looks at the long-term safety and effectiveness of a drug called mexiletine for people with Myotonic Dystrophy Type 1 or Type 2. It's for those who have already completed a previous study with this medication.
Belgium · Denmark · 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