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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)

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Trial Profile

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: Discontinued
Phase of Trial: Phase III

Latest Information Update: 13 Oct 2025

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At a glance

  • Drugs Epalrestat (Primary)
  • Indications Congenital disorder of glycosylation type 1A
  • Focus Therapeutic Use

Most Recent Events

  • 13 Oct 2025 Last checked against ClinicalTrials.gov: US National Institutes of Health record.
  • 25 Sep 2025 Status changed from active, no longer recruiting to discontinued due to futility.
  • 30 May 2024 Planned primary completion date changed from 28 Feb 2024 to 28 Feb 2025.

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