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)

Status: Not yet recruiting

Phase of Trial: Phase II

Latest Information Update: 28 Mar 2025

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At a glance

Drugs D-galactose (Primary)
Indications Glycogen storage disease XIV; Phosphoglucomutase 1 deficiency
Focus Therapeutic Use

Most Recent Events

25 Mar 2025 Planned initiation date changed from 1 Mar 2025 to 1 Sep 2025.
30 Dec 2024 Planned initiation date changed from 1 Dec 2024 to 1 Mar 2025.
16 Oct 2024 Primary endpoint changed from 'Time (days) to rescue treatment from randomization [Time Frame: 16 weeks]' to ' Proportion of participants with at least one PGM1-CDG related event [Time Frame: after each treatment period (treatment periods are 18 weeks)]

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