Phase
Condition
Kidney Disease
Fabry Disease
Treatment
ERT
migalastat HCl
Clinical Study ID
Ages > 18 All Genders
Study Summary
Eligibility Criteria
Inclusion
I. Migalastat-treated patients (Commercial only participants)
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
Patients who show a decline in their Fabry disease symptomatology based on any of the following:
a decrease in annualized rate of decline eGFRCKD-EPI of ≥ 2 mL/min/1.73 m2 during the 2 years prior to enrollment
microalbuminuria/macroalbuminuria (≥ 30 mg/24 h or ≥ 20 mg on first morning urine) or urine ACR of ≥ 30 mg/g (via spot urine collection) at any time prior to or at enrollment
proteinuria (> 0.5 g/g UPCR) any time prior to or at enrollment
males with classic Fabry disease phenotype
II. Migalastat-treated patients who are not considered to be in renal decline (Commercial migalastat users only)
- Patients with Fabry disease with amenable GLA variants who have been on commercial migalastat regardless of the duration of treatment
III. Migalastat-treated patients (Prior clinical trial participants)
- Patients with Fabry disease 18 years or older who had commenced treatment with migalastat while in a clinical trial and were exposed to treatment for at least 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 who are still taking migalastat at the time of enrollment, having switched to commercial product
IV. Untreated patients
Patients with Fabry disease 18 years or older with amenable GLA variants, who have never been on treatment for Fabry disease, who have an eGFR greater than or equal to 30 mL/min/1.73 m2 at the time of enrollment, and who meet local treatment guidelines for Fabry disease
Patients who show a decline in their Fabry disease symptomatology based on any of the following:
a decrease in annualized rate of decline eGFRCKD-EPI of ≥ 2 mL/min/1.73 m2 during the 2 years prior to enrollment
microalbuminuria/macroalbuminuria (≥ 30 mg/24 h or ≥ 20 mg on first morning urine) or urine ACR of ≥ 30 mg/g (via spot urine collection) at any time prior to or at enrollment
proteinuria (> 0.5 g/g UPCR) any time prior to or at enrollment
males with classic Fabry disease phenotype
V. ERT-treated patients
Patients with Fabry disease 18 years or older who have commenced ERT 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 being treated with ERT at the time of enrollment, and who have amenable GLA variants
Patients who show a decline in their Fabry disease symptomatology based on any of the following:
a decrease in eGFRCKD-EPI annualized rate of decline of ≥ 2 mL/min/1.73 m2 during the 2 years prior to enrollment
microalbuminuria/macroalbuminuria (≥ 30 mg/24 h or ≥ 20 mg on first morning urine) or urine ACR of ≥ 30 mg/g (via spot urine collection) at any time prior to or at enrollment
proteinuria (> 0.5 g/g UPCR) any time prior to or at enrollment
males with classic Fabry disease phenotype
All patients 1. All treated and untreated patients with Fabry disease who are enrolled in the study must be able to understand and provide written informed consent or assent.
Exclusion Criteria
- Patients who currently are participating in a clinical trial of any investigational medicinal product or device at the time of enrollment
Study Design
Study Description
Connect with a study center
UAB Nephrology Research Clinic at Paula Building
Birmingham 4049979, Alabama 4829764 35233
United StatesSite Not Available
Arkansas Children's Hospital
Little Rock 4119403, Arkansas 4099753 72202
United StatesSite Not Available
Emory Genetics
Atlanta, Georgia 30322
United StatesSite Not Available
Emory Genetics
Atlanta 4180439, Georgia 4197000 30322
United StatesActive - Recruiting
Washington University School of Medicine
St. Louis, Missouri 63110
United StatesSite Not Available
Washington University School of Medicine
St Louis 4407066, Missouri 4398678 63110
United StatesSite Not Available
UPMC Children's Hospital of Pittsburgh
Pittsburgh 5206379, Pennsylvania 6254927 15224
United StatesSite Not Available
Renal Disease Research Institute
Dallas, Texas 75204
United StatesSite Not Available
Renal Disease Research Institute
Dallas 4684888, Texas 4736286 75204
United StatesActive - Recruiting
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia 22030
United StatesSite Not Available
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax 4758023, Virginia 6254928 22030
United StatesActive - Recruiting

Not the study for you?
Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.