Natural History and Structural Functional Relationships in Fabry Renal Disease Natural History, Structural Functional Relationships and Determinants of Renal Structural Responses (Changes) With Enzyme Replacement Therapy in Fabry Disease

To hypothesis that mosaicism in Fabry disease females is such that cells such as podocytes are either affected or normal and that ERT clearance in the affected podocytes in females will, as in males, be dependant on age of institution of treatment and ERT dose.

CT.gov Identifier
NCT01581424
EudraCT Identifier
N/A
CTIS:
N/A
Sponsor
Washington University School of Medicine
Collaborator
National Institutes of Health/National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health/National Institute of Neurological Disorders and Stroke, (Other Hospital/Academic/Medical Center), University of Minnesota
Study Contact Information
N/A
Recruiting
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Study Details

Diseases
Fabry Disease
Study Drug
Enzyme replacement therapy, unspecified
Genes
GLA
Study Dates
Oct 2010 - Jul 2030
Sex
Female & Male
Age
1 - 75 Years
Inclusion and Exclusion Criteria
Inclusion Criteria:
  • Patients diagnosed with Fabry disease who have/have not received enzyme replacement therapy where a clinical decision has been made to obtain a kidney biopsy, a GFR, and urinary albumin studies or where patients have previously completed clinical trials which included measures of renal function and renal biopsies.
Exclusion Criteria:
  • Patients with serum creatinine more than 2.5 mg/dL or known to have a renal disease other than Fabry.

Protocol Summary

To hypothesis that mosaicism in Fabry disease females is such that cells such as podocytes are either affected or normal and that ERT clearance in the affected podocytes in females will, as in males, be dependant on age of institution of treatment and ERT dose.

Study Locations

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