Ignite Creation Date:
2024-05-06 @ 4:15 PM
Last Modification Date:
2024-10-26 @ 2:06 PM
Study NCT ID:
NCT04925960
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-06-03
First Post:
2021-06-08
Brief Title:
Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG
Sponsor:
Maggies Pearl LLC
Organization:
Maggies Pearl LLC
Study Overview
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
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
Detailed 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None