NCT05402332View on ClinicalTrials.gov

Evaluating the Efficacy and Safety of D-galactose in PGM1-CDG (AVTX-801)

PHASE2Not yet recruitingINTERVENTIONAL
Enrollment

8

Participants

Timeline

Start Date

February 2, 2026

Primary Completion Date

March 2, 2028

Study Completion Date

March 2, 2028

Conditions
PGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of Glycosylation
Interventions
DRUG

AVTX-801

D-Galactose medical grade supplement - 1.5g/kg/day (not to exceed 50 g/day)

DRUG

Placebo

placebo equivalent

Trial Locations (1)

55905

Mayo Clinic Rochester, Rochester

All Listed Sponsors
collaborator

Children's Hospital of Philadelphia

OTHER

lead

Eva Morava-Kozicz

OTHER