The study will be a randomized, double blind, placebo-controlled study of the safety and efficacy of PRX-102 in ERT naïve male patients randomized 1:1. Patient age will be 14 to 45 years. Patients must have diarrhea defined as ≥ 3 stools a day with an average consistency of ≥ 5.5 on the Bristol Stool Form Scale (BSFS) by patient electronic diary and moderate to severe gastrointestinal symptoms as defined by the Irritable Bowel Symptom Severity Score (IBSSS) Part 1 average \> 175 derived from at least two IBSSS assessments during screening period. Patients will receive intravenous infusions of PRX-102 1 mg/kg or placebo every two weeks for 6 months.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
IBSSS Part 1
Irritable Bowel Syndrome Severity Score
Time frame: Every 2 weeks for 6 months
Stool frequency
from BSFS diary
Time frame: After every bowel movement for 6 months
Body Weight
Time frame: Every 2 weeks for 6 months
Plasma Lyso-Gb3
Time frame: Every 4 weeks for 6 months
Plasma Gb3
Time frame: Every 4 weeks for 6 months
Urine Lyso-GB3
Time frame: Every 6 weeks for 6 months
Frequency of pain medication use
Time frame: Every 2 weeks for 6 months
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