Phase 2 Myotonic Dystrophy Clinical Trials
8 recruitingPhase 2
What is a Phase 2 trial?
Phase 2 trials evaluate whether a treatment works for a specific condition and further assess its safety. These studies typically enroll several hundred participants and provide preliminary data on effectiveness.
Showing 1–8 of 8 trials
Recruiting
Phase 1Phase 2
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
Myotonic Dystrophy
Sanofi32 enrolled9 locationsNCT06844214
Recruiting
Phase 2
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Myotonic Dystrophy 1
PepGen Inc24 enrolled8 locationsNCT06667453
Recruiting
Phase 2
An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
Myotonic Dystrophy 1
PepGen Inc48 enrolled3 locationsNCT07220603
Recruiting
Phase 1Phase 2
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1)
Dyne Therapeutics116 enrolled17 locationsNCT05481879
Recruiting
Phase 1Phase 2
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Myotonic Dystrophy Type 1 (DM1)
Vertex Pharmaceuticals Incorporated44 enrolled26 locationsNCT06185764
Recruiting
Phase 1Phase 2
Study of ATX-01 in Participants With DM1
Myotonic Dystrophy 1
ARTHEx Biotech S.L.56 enrolled12 locationsNCT06300307
Recruiting
Phase 1Phase 2
Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
Myotonic Dystrophy 1
Arrowhead Pharmaceuticals78 enrolled11 locationsNCT06138743
Recruiting
Phase 2Phase 3
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
Congenital Myotonic Dystrophy
AMO Pharma Limited76 enrolled14 locationsNCT05004129