Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG (NCT04925960) | Clinical Trial Compass
TerminatedPhase 3
Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG
Stopped: Due to futility
United States42 participantsStarted 2022-11-10
Plain-language 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.
Who can participate
Age range2 Years – 17 Years
SexALL
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Inclusion criteria
✓. Age ≥ 2 and \< 18 years
✓. Diagnosis of PMM2-CDG, based on molecularly confirmed biallelic PMM2 pathogenic variants (can be historical diagnosis with lab report on file)
✓. Informed consent (and assent, as applicable) document personally signed by the legally authorized representative of the patient, indicating that the patient's parent/guardian has been informed and agreed to all aspects of the study
✓. Be willing and able to adhere to the study assessments and schedule described in the protocol and consent/assent documents
✓. Negative urine pregnancy test (only for female subjects of child-bearing potential)
✓. For subjects of child-bearing potential-only, subject has been counseled on and agrees to the requirement either for double barrier contraceptive methods and/or for total abstinence from prior to randomization through 3-months after the cessation of treatment.
Exclusion criteria
✕. Known or suspected other known CDG
✕. Known allergy to aldose reductase inhibitors
✕. Hypersensitivity to epalrestat
✕. Hepatic impairment defined as any one of the following:
✕. AST/ALT \>5x ULN in the 6 months prior to screening
✕. Bilirubin \>2X ULN in the last 6 months prior to screening