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

Part of paid clinical trials in Rochester, Minnesota.

Sponsor
Eva Morava-Kozicz
Study ID
NCT05402332
Phase
PHASE2
Status
Not Yet Recruiting

Notify me when recruiting opens

Save your spot on the interest list for this study. We'll keep your details with this study so our team can follow up when recruiting opens.

Not yet recruiting

Add your contact details and location so we can keep your interest tied to this study.

Conditions

  • PGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of Glycosylation

Eligibility Criteria

Sex
ALL
Age
18 Years - 60 Years
Healthy Volunteers
Not accepted

Interventions

  • AVTX-801 — DRUG
    D-Galactose medical grade supplement - 1.5g/kg/day (not to exceed 50 g/day)
  • Placebo — DRUG
    placebo equivalent

Study Details

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.

Key Dates

Start date
Oct 1, 2026
Status verified
Mar 2026
Primary completion
Mar 2, 2028
Completion
Mar 2, 2028

Study Design

Enrollment
8 participants (estimated)
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Experimental: AVTX-801, then Placebo
    Participants receive AVTX-801 1.5g/kg/day (in applesauce) during Treatment Period 1 and then placebo (in applesauce) during Treatment Period 2.
  • Experimental: Placebo, then 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.

Primary Outcome Measure

Proportion of participants with at least one PGM1-CDG related event [ Time Frame: after each treatment period (treatment periods are 18 weeks) ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Mayo Clinic RochesterRochesterMinnesota55905-

Find similar trials in Rochester, MN

By research site
Mayo Clinic Rochester· Rochester, MN