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
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
10
Mayo Clinic Minnesota
Rochester, Minnesota, United States
Number of major motor seizure frequency
Seizure burden assessed by change from baseline in 28-day major motor seizure frequency
Time frame: 28 days
Number of vomiting episodes
Vomiting frequency in 28-day from baseline
Time frame: 28 days
Bristol Stool Form Scale (BSFS)
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.
Time frame: average daily 28 days
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