Observational Study Evaluating Long-Term Effects and Safety of Migalastat and Enzyme Replacement Therapy in Adults with Fabry Disease in the US

What this Trial Is About

Researchers are conducting a global observational study to assess the long-term effects of treatments on patients with Fabry disease, focusing mainly on the drug migalastat. The study aims to evaluate the safety, effectiveness, and health-related quality of life among patients receiving different treatments or no treatment at all. This study plans to enroll at least 450 patients, including those treated with migalastat, enzyme replacement therapy ERT, and untreated individuals who have never received therapy for Fabry disease. Participants will be grouped based on their treatment status those treated with migalastat within 24 months before enrollment, those receiving ERT within the same period, and untreated patients meeting criteria for migalastat treatment. Enrollment will last for 5 years, and all patients will be followed for up to 5 years after joining. The study is non-interventional, meaning patients will continue their usual care while researchers collect data on their condition and treatment effects. During the study, participants will provide information through medical assessments and questionnaires to track kidney function, clinical events related to Fabry disease, enzyme activity, pain levels, gastrointestinal symptoms, and quality of life over time. Researchers will also monitor safety events and overall survival. Data will be collected retrospectively and prospectively, covering up to 5 years. The studys main measure is the change in kidney function rate. Participants can expect periodic evaluations to understand how treatments impact their health and well-being over the long term.

A Study of Patients With Fabry Disease (US Specific)