Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG

Pmm2-CDG Clinical Trial

Official title: A Prospective, Randomized, Double-Blind, Placebo-Controlled, Single-Center Study of Oral Epalrestat Therapy in Pediatric Subjects With Phosphomannomutase 2-congenital Disorder of Glycosylation (PMM2-CDG)

Status Active, not recruiting

Clinical Trial 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.

Clinical Trial Description

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. The primary study objective is to evaluate the safety and probable benefit of oral epalrestat therapy in pediatric subjects with PMM2-CDG. Study outcomes include evaluating the metabolic improvement of pediatric subjects treated with oral epalrestat therapy compared to placebo, evaluating safety, clinical improvement, and pharmocokinetics (PK) of oral epalrestat therapy in pediatric subjects compared to placebo, and evaluating urine polyols, adverse events, laboratory data, other safety measures, PK, and Quality of Life surveys to measure clinical improvement. ;

Related Conditions & MeSH terms

• Congenital Disorders of Glycosylation
• Phosphomannomutase 2 Deficiency
• Pmm2-CDG

• NCT number: NCT04925960
• Study type: Interventional
• Source: Maggie's Pearl, LLC
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: November 10, 2022
• Completion date: December 31, 2025