PGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of Glycosylation Clinical Trial
Official title:
A Phase 2b, Multicenter, Randomized, Double-blind, Placebo-Controlled, Crossover, Trial Assessing the Efficacy, Safety, and Tolerability of AVTX-801 in Subjects With Phosphoglucomutase 1 Deficiency Related Congenital Disorders of Glycosylation (PGM1-CDG)
You have been asked to take part in this research study because you have been diagnosed with PGM1-CDG and are currently taking D-galactose.
Upon randomization, subjects will be randomly assigned to either AVTX-801 1.5 g/kg/day (not to exceed 50 g/day) or blinded withdrawal for 16 weeks. Efficacy will be assessed from the Baseline Visit (Day 1) to the End of Double-blind Period Visit (Day 112). Subjects who are D-galactose treatment-naïve may enter the randomized, double blind portion of the study after completing at least 6 weeks of stable doses of AVTX-801 at 1.5 g/kg/day. Dosing with AVTX-801 will begin with 0.25 g/kg/day for at least 14 days. Doses may be increased every 14 days at the following schedule: 0.5 g/kg/day, 1.0 g/kg/day, and 1.5 g/kg/day. Dose escalation will be based on the Investigator's assessment of individual subject tolerability and improvement in the key clinical laboratory parameters (ATIII, creatine kinase [CK], ALT, AST, and fasting blood glucose). Maximum daily dose should not exceed 50 g/kg/day. Once the Investigator has determined a safe, effective, and well tolerated dose, a 6-week run in period will begin with the selected dose. Upon completion of the 6-week run-in period, subject will be randomized and proceed into the study. During the randomized portion of the study, subjects will be closely monitored for clinical signs and symptoms related to or suspected to be related to withdrawal of D galactose therapy; specifically, recurrent or prolonged hypoglycemia, prolonged elevation of ALT, and decreases in ATIII. ;