Paricalcitol in Fabry Disease

March 17, 2014 updated by: Eleonora Riccio, Federico II University
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

14

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Naples, Italy, 80129
        • federico II university, department of nephrology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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
Drug: Paricalcitol
Paricalcitol was administered at the dose of 1 mcg/die

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Effect of paricalcitol on proteinuria reduction
Time Frame: 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.
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Federico II University

Investigators

  • Principal Investigator: eleonora riccio, md, Federico II University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2012

Primary Completion (Actual)

December 1, 2013

Study Completion (Actual)

December 1, 2013

Study Registration Dates

First Submitted

March 14, 2014

First Submitted That Met QC Criteria

March 17, 2014

First Posted (Estimate)

March 18, 2014

Study Record Updates

Last Update Posted (Estimate)

March 18, 2014

Last Update Submitted That Met QC Criteria

March 17, 2014

Last Verified

January 1, 2014

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PCT-FD

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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