Paricalcitol in Fabry Disease

NCT ID: NCT02090608

Last Updated: 2014-03-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-03-31
• Study Completion Date: 2013-12-31

Brief 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.

Conditions

Fabry Disease; Proteinuria

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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

Group Type: EXPERIMENTAL

Paricalcitol

Intervention Type: DRUG

Paricalcitol was administered at the dose of 1 mcg/die

Interventions

Paricalcitol

Paricalcitol was administered at the dose of 1 mcg/die

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Federico II University

OTHER

Sponsor Role: lead

Responsible Party

Eleonora Riccio

MD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

eleonora riccio, md

Role: PRINCIPAL_INVESTIGATOR

Federico II University

Locations

federico II university, department of nephrology

Naples, Naples, Italy

Countries

Italy

Other Identifiers

PCT-FD

Identifier Type: -

Identifier Source: org_study_id