Paricalcitol in Fabry Disease

Fabry Disease Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02090608
• Other study ID #: PCT-FD
• Status: Completed
• Phase: N/A
• First received: March 14, 2014
• Last updated: March 17, 2014
• Start date: March 2012
• Est. completion date: December 2013

Study information

• Verified date: January 2014
• Source: Federico II University
• Contact: n/a
• Is FDA regulated: No
• Health authority: Italy: Ethics Committee
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: December 2013
• Est. primary completion date: December 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 75 Years

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fabry Disease
• Proteinuria

Intervention

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

Locations

Country	Name	City	State
Italy	federico II university, department of nephrology	Naples

Sponsors (1)

Lead Sponsor	Collaborator
Federico II University

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Effect of paricalcitol on proteinuria reduction	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.	6 months	No