drugset / Trial / NCT03692312
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
RandomizedParallel-groupQuadruple-blindTreatment
Summary
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
Timeline
- Start
- 2021-03-03
- Primary completion
- 2023-04-04
- Completion
- 2023-04-04
Outcome
Missed primary endpoint
registry analysis (superiority test); Tideglusib vs Placebo; p = 0.0514; Mean Difference (Final Values) 1.75 (95% CI -0.01 to 3.51); MMRM NCT03692312 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Tideglusib | Small molecule | 400 mg | Oral |
| Subject | Tideglusib | Small molecule | 600 mg | Oral |
| Subject | Tideglusib | Small molecule | 1000 mg | Oral |