Paricalcitol in Fabry Disease

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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Key information

Conditions

Fabry Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Genetic Diseases, X-Linked Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Pathological Conditions, Signs and Symptoms Proteinuria Signs and Symptoms Sphingolipidoses Urination Disorders Urogenital Diseases Urologic Diseases Urological Manifestations Vascular Diseases

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

federico II university, department of nephrology

Naples, 80129, Italy

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Paricalcitol

Drug

Paricalcitol was administered at the dose of 1 mcg/die

Primary outcomes

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.

Sponsors and collaborators

Lead sponsor

Federico II University

Other

Registry information

Important dates

Study start: 2012
Primary completion: 2013
Study completion: 2013
First posted: Mar 18, 2014
Registry last updated: Mar 18, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed