A Prospective, Observational Study of Patients With Fabry Disease (US Specific)
Amicus Therapeutics
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.
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 sit…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
I. Migalastat-treated patients (Commercial only participants) 1. Patients with Fabry disease 18 years or older with amenable GLA variants who have commenced commercial migalastat treatment within 24 months preceding enrollment, who have an eGFR greater than or equal to 30 mL/min/1.73 m2 at the time of enrollment and are still taking migalastat at the time of enrollment, or who are starting migalastat at the time of enrollment, excluding those who participated in a prior migalastat clinical trial 2. Patients who show a decline in their Fabry disease symptomatology based on any of the following…
Interventions
- Drugmigalastat HCl
Non-interventional study of participants receiving migalastat HCl 150 mg
- DrugERT
Non-interventional study of participants receiving enzyme replacement therapy
Locations (8)
- UAB Nephrology Research Clinic at Paula BuildingBirmingham, Alabama
- Arkansas Children's HospitalLittle Rock, Arkansas
- Emory GeneticsAtlanta, Georgia
- Washington University School of MedicineSt Louis, Missouri
- New York-Presbyterian Morgan Stanley Children's Hospital - Columbia University Medical CenterNew York, New York
- UPMC Children's Hospital of PittsburghPittsburgh, Pennsylvania