Clinical Trial

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

Study acronym: AVTX-801
Not Yet Recruiting Phase 2
View on ClinicalTrials.gov →
Summary
This is a clinical trial to evaluate the efficacy of AVTX-801 (D-galactose) on the clinical manifestations of PGM1-CDG in participants currently taking D-galactose.
Protocol Amendment History 10 amendments
This ClinicalTrials.gov record has been amended 10 times since 2022-05-28; most recent amendment 2026-03-12.
Trial Details
NCT Number NCT05402332
Lead Sponsor Eva Morava-Kozicz
Collaborators: Children's Hospital of Philadelphia
Conditions PGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of Glycosylation
Enrollment 8 participants
Start Date 2026-10-01
Primary Completion 2028-03-02 (estimated)
Study Completion 2028-03-02 (estimated)
Updated on ClinicalTrials.gov 2026-03-16