Paricalcitol in Fabry Disease

Status

Completed

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02090608

Enrollment

Registered

2014-03-18

Start date

2012-03-31

Completion date

2013-12-31

Last updated

2014-03-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fabry Disease, Proteinuria

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.

Interventions

DRUGParicalcitol

Paricalcitol was administered at the dose of 1 mcg/die

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 75 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
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.

Countries

Italy

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026