NCT02921620

Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease

A Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease Patients

• Status: Withdrawn
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Protalix · Industry
• Sex: Male
• Age: 14 Years – 45 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
BIOLOGICAL	PRX-102	Intravenous
OTHER	Placebo	Intravenous

Timeline

• Start date: 2017-07-01
• Primary completion: 2018-07-01
• Completion: 2018-07-01
• First posted: 2016-10-03
• Last updated: 2018-01-08

Regulatory

• FDA-regulated drug study