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4 recruiting studies matching your filters
Phase 1
A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with My…
Myotonic Dystrophy Type 1 (DM1)
Dyne TherapeuticsNCT05481879
Phase 3
A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1…
Myotonic Dystrophy Type 1 (DM1)DM1Myotonic Dystrophy+2 more
Dyne TherapeuticsNCT07486934
Phase 1
A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in pa…
Myotonic Dystrophy Type 1 (DM1)
Vertex Pharmaceuticals IncorporatedNCT06185764
REACH DM - Remote Assessments and Genetic Determinants of Myotonic Dystrophy
The goal of this observational study, conducted in participants' homes and requiring no travel to a study site, is to better understand disease variability in …
Myotonic Dystrophy Type 1 (DM1)DM1
University of RochesterNCT07505342