Paricalcitol in Fabry Disease

Summary

Proteinuria is the predominant risk factor for renal disease progression in Fabry disease (FD). When urine protein excretion is controlled to \<0.50 g/24 hr, the rate loss of glomerular filtration rate (GFR) is not significantly different from 0. However, enzyme replacement therapy (ERT) alone does not decrease proteinuria and it has been recommended that patients receiving ERT also receive anti Renin-Angiotensin-System (RAS) therapy. Emerging evidences show that paricalcitol (PCT) reduces proteinuria in presence of intensified inhibition of RAS; however, there is no evidence in FD. The aim of this study is to evaluate the antiproteinuric effect of PCT in FD patients with proteinuria \>0.50 g/24 hr persisting despite the ERT and anti-RAS therapy titrated to maximum tolerated dosage.

Timeline

Start

2012-03

Primary completion

2013-12

Completion

2013-12

Drugs

Indications