Open-Label Phase IV Study to Assess the Safety and Effectiveness of Replagal Infusions Every Two Weeks in Indian Children and Adults With Fabry Disease

What this Trial Is About

Researchers are evaluating the safety and effectiveness of Replagal in children and adults with Fabry disease in India. This open-label, single-arm Phase IV study aims to better understand the safety profile of Replagal by monitoring participants for about one year. Fabry disease participants must have a confirmed diagnosis based on gene mutation or enzyme deficiency and show clinical symptoms. Participants will receive Replagal at a dose of 0.2 mg per kilogram through intravenous infusion starting on Day 1 and then every two weeks up to Week 51. The treatment is given at the clinic and is designed to assess safety and potential benefits over approximately one year. This study does not include a comparison group and focuses on the effects of Replagal alone. During the study, participants will have regular assessments including kidney function tests, heart measurements, urine analyses, and quality of life questionnaires at various time points up to Week 53. Researchers will monitor adverse events, infusion reactions, and any treatment-related side effects throughout the study. Participants will be followed closely with scheduled visits to evaluate safety and any changes in their condition over the course of the year.

A Study of Replagal in Children and Adults With Fabry Disease in India