Phase 1 Myotonic Dystrophy Clinical Trials
5 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–5 of 5 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 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