AVTX-801 D-galactose Supplementation in SLC35A2-CDG
Phase 2
Not yet recruiting
- Conditions
- SLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of Glycosylation
- Interventions
- Drug: Placebo
- Registration Number
- NCT05402384
- Lead Sponsor
- Eva Morava-Kozicz
- Brief Summary
This is a multicenter, randomized, double-blind, placebo-controlled, cross-over study to evaluate the efficacy and safety of AVTX-801 in subjects with SLC35A2-CDG
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 10
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description AVTX-801, then Placebo AVTX-801 Each treatment period is 24 weeks, with 6-week washout period in between. AVTX-801, then Placebo Placebo Each treatment period is 24 weeks, with 6-week washout period in between. Placebo, then AVTX-801 AVTX-801 Each treatment period is 24 weeks, with 6-week washout period in between. Placebo, then AVTX-801 Placebo Each treatment period is 24 weeks, with 6-week washout period in between.
- Primary Outcome Measures
Name Time Method Number of major motor seizure frequency 28 days Seizure burden assessed by change from baseline in 28-day major motor seizure frequency
Number of vomiting episodes 28 days Vomiting frequency in 28-day from baseline
Bristol Stool Form Scale (BSFS) average daily 28 days Constipation/diarrhea assessed by scale improvement toward normal stool forms from baseline in average daily 28-day Bristol Stool form scale. The BSFS classifies stool into 7 groups, type 1 (hardest) to type 7 (softest). Type 3 and Type 4 would be more indicative of normal stool.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Mayo Clinic Minnesota
🇺🇸Rochester, Minnesota, United States