NCT06906367

A Study of Patients With Fabry Disease (US Specific)

A Prospective, Observational Study of Patients With Fabry Disease (US Specific)

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 450 (estimated)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is an observational study to evaluate the effects of treatment on long-term effectiveness, safety, and health-related quality of life (HRQOL) in patients with Fabry disease, with a main focus on migalastat.

Detailed description

This is a prospective, multicenter, observational, effectiveness, safety, and outcomes study enrolling at least 450 patients with Fabry disease globally (at least 250 patients in the migalastat-treated group, approximately 100 patients in the ERT-treated group, and approximately 100 patients in the untreated group \[patients who have never been on treatment for Fabry disease\]). Enrollment will continue for a period of 5 years and all patients will be followed for up to 5 years after their enrollment. Disclaimer: This is a global study, the country level requirements may vary from site to site. The requirements noted in this posting are specific to the US.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	migalastat HCl	Non-interventional study of participants receiving migalastat HCl 150 mg
DRUG	ERT	Non-interventional study of participants receiving enzyme replacement therapy

Timeline

• Start date: 2026-02-13
• Primary completion: 2032-06-01
• Completion: 2032-06-01
• First posted: 2025-04-02
• Last updated: 2026-03-13

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study