A Study of Patients With Fabry Disease (US Specific)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

450 participants

Study Classification

OBSERVATIONAL

Study Start Date

2026-01-31

Study Completion Date

2032-06-30

Brief Summary

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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.

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Detailed Description

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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

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Fabry Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Migalastat-treated

Migalastat-treated patients at the time of enrollment who started the treatment within the 24 months prior to enrollment.

migalastat HCl

Intervention Type DRUG

Non-interventional study of participants receiving migalastat HCl 150 mg

ERT-treated

Patients receiving ERT at the time of enrollment who started the treatment within the 24 months prior to enrollment.

ERT

Intervention Type DRUG

Non-interventional study of participants receiving enzyme replacement therapy

Untreated

Untreated patients at the time of enrollment; these patients must never have been on therapy for Fabry disease prior to enrollment into the study and must meet criteria for receiving treatment with migalastat.

No interventions assigned to this group

Interventions

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migalastat HCl

Non-interventional study of participants receiving migalastat HCl 150 mg

Intervention Type DRUG

ERT

Non-interventional study of participants receiving enzyme replacement therapy

Intervention Type DRUG

Eligibility Criteria

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Exclusion Criteria

1\. Patients who currently are participating in a clinical trial of any investigational medicinal product or device at the time of enrollment

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Research

Role: STUDY_DIRECTOR

Amicus Therapeutics

Locations

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