Paricalcitol in Fabry Disease

NCT02090608 — Not applicable, Completed, 14 participants, Federico II University

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Registry	NCT02090608
Status	Completed
Phase	Not applicable
Study type	Interventional; non randomized, single group, masking: none, treatment
Enrollment	14 (Actual)
Sponsor	Federico II University (Other)
Conditions	Fabry Disease, Proteinuria
Interventions	Paricalcitol (Drug)
Dates	start 2012-03-01, primary completion 2013-12-01, completion 2013-12-01, first posted 2014-03-18, last updated 2014-03-18
Locations	1 site in Italy

-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.

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+Arms and interventions

Arm	Type	Interventions	Description
Paricalcitol	Experimental	Drug: Paricalcitol	In patients identified by the inclusion criteria, data will be collected at baseline , during administration of oral Paricalcitol (PCT) (after 1, 3 and 6 months), and three months after PCT withdrawal. PCT will administered at dosage of 1 mcg/day; this dosage was chosen as it is not associated with excessive decline of parathyroid hormone (PTH) levels in most patients

Paricalcitol (Drug): Paricalcitol was administered at the dose of 1 mcg/die

+Outcome measures

Primary outcomes

Effect of paricalcitol on proteinuria reduction [6 months]: Fourteen Fabry patients will be selected and studied in the first six months of add-on oral PCT (1 mcg/day) and, in order to verify the dependence of proteinuria reduction on PCT, three months after drug withdrawal.

+Eligibility

Sex: All, ages: 18 Years to 75 Years

Inclusion Criteria:

* genetically proven FD
* stable dose of ERT for at least 12 months
* stable dose of ACEi or ARB titrated to maximum tolerated dosage for at least 6 months
* persistent proteinuria >0.50 g/24 h despite the use of ERT and ACEi/ARBs in 2 consecutive samples within 12 weeks

Exclusion Criteria:

* steroid/immunosuppressive treatment or glomerular filtration rate change >30% in the past 3 months
* PTH levels <20 pg/mL
* serum phosphorus >5.0 mg/dL
* serum calcium (adjusted for albumin) >10.0 mg/dL
* active malignancy.

+Locations (1)

Facility	City	Country	Status
federico II university, department of nephrology	Naples, Naples	Italy

+Investigators

eleonora riccio, md, Principal investigator, Federico II University

My Notes

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