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)
Eva Morava-Kozicz
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.
Description
Participants who sign consent will be screened for enrollment, and if they meet all eligibility criteria, they will enter a 4-week Run-In period. During the Run-In period, participants will continue to take D-galactose therapy, and will be monitored for hypoglycemic events, to establish a baseline rate. Following completion of the Run-In period participants will complete Baseline Day 1 procedures for treatment period 1 and be randomized in a 1:1 fashion to one of two treatment sequences: 1) AVTX-801 (plus applesauce) 1.5 g/kg/day (not to exceed 50 g/day) (treatment period 1) followed by placeb…
Eligibility
- Age range
- 18–60 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria 1. Subject is aged 18 to 60 years. 2. Subject has biologically and genetically proven PGM1-CDG. 3. Subject is currently on a stable dose of D-galactose therapy. 4. Non-pregnant, non-lactating female subjects of childbearing potential who are heterosexually active and non-sterile male subjects with female sexual partners of childbearing potential agree to use a highly effective method of contraception for the duration of the study, including the long-term safety follow-up period. A highly effective method of birth control is defined as one that results in a low failure rate…
Interventions
- DrugAVTX-801
D-Galactose medical grade supplement - 1.5g/kg/day (not to exceed 50 g/day)
- DrugPlacebo
placebo equivalent
Location
- Mayo Clinic RochesterRochester, Minnesota