drugset / Trial / NCT04925960
Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG
RandomizedParallel-groupQuadruple-blindTreatment
Summary
This is a prospective, single-center, randomized, double-blind, placebo-controlled study designed to assess the safety, tolerability, and clinical and metabolic improvement of pediatric subjects with PMM2-CDG on oral epalrestat therapy vs. placebo.
Timeline
- Start
- 2022-11-10
- Primary completion
- 2025-02-28
- Completion
- 2025-02-28
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Epalrestat | Small molecule | — | Oral |