Paricalcitol in Fabry Disease
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 14
- Locations
- 1
- Primary Endpoint
- Effect of paricalcitol on proteinuria reduction
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 75 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
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
Intervention: Paricalcitol (Drug)
Outcomes
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.
Secondary Outcomes
No secondary outcomes reported
Investigators
Eleonora Riccio
MD
Federico II University