Evaluating the Efficacy and Safety of D-galactose in PGM1-CDG (AVTX-801)
- Conditions
- PGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of Glycosylation
- Interventions
- Drug: Placebo
- Registration Number
- NCT05402332
- Lead Sponsor
- Eva Morava-Kozicz
- Brief 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.
- Detailed 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 placebo (plus applesauce ) (treatment period 2) or 2) Placebo (plus applesauce) (treatment period 1) followed by AVTX-801 (plus applesauce) 1.5 g/kg/day (not to exceed 50 g/day) (treatment period 2).
Each treatment period will end upon completion of 18 weeks of treatment or upon occurrence of a PGM1-CDG related event. There will be an open label recovery period of 18 weeks separating the two treatment periods, during which time the subject will receive commercially available D-galactose.
During the double-blind period of the study, participants will be closely monitored for clinical signs and symptoms related to or suspected to be related to withdrawal of D galactose therapy; specifically, recurrent or prolonged hypoglycemia, elevation of ALT and decreases in ATIII.
Upon completion of the double-blind portion of the study (i.e., either completion of both 18-week double-blind periods or occurrence of a PGM1-CDG related event during treatment period 2), participants will be permitted to enter a long-term, open-label, safety follow-up period of 12 months with AVTX-801.
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 8
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description AVTX-801, then Placebo AVTX-801 Participants receive AVTX-801 1.5g/kg/day (in applesauce) during Treatment Period 1 and then placebo (in applesauce) during Treatment Period 2. AVTX-801, then Placebo Placebo Participants receive AVTX-801 1.5g/kg/day (in applesauce) during Treatment Period 1 and then placebo (in applesauce) during Treatment Period 2. Placebo, then AVTX-801 AVTX-801 Participants receive placebo (in applesauce) during Treatment Period 1 and then AVTX-801 1.5g/kg/day (in applesauce) during Treatment Period 2. Placebo, then AVTX-801 Placebo Participants receive placebo (in applesauce) during Treatment Period 1 and then AVTX-801 1.5g/kg/day (in applesauce) during Treatment Period 2.
- Primary Outcome Measures
Name Time Method Proportion of participants with at least one PGM1-CDG related event after each treatment period (treatment periods are 18 weeks) Proportion of participants in each treatment group who experience at least one PGM1-CDG related event (recurrent or prolonged hypoglycemia, elevation of ALT and/or decrease in ATIII) during each treatment period.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Mayo Clinic Rochester
🇺🇸Rochester, Minnesota, United States