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

NCT05402332 · Status: NOT_YET_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2026-03-16

No results posted yet for this study

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.

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

Sponsors & Collaborators

Principal Investigators

  • Eva Morava-Kozicz, MD, PhD · Icahn School of Medicine at Mount Sinai

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
CROSSOVER

Eligibility

Min Age
18 Years
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-10-01
Primary Completion
2028-03-02
Completion
2028-03-02
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05402332 on ClinicalTrials.gov