Paricalcitol in Fabry Disease
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Effect of paricalcitol on proteinuria reduction
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
研究组 & 干预措施
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
干预措施: Paricalcitol (Drug)
结局指标
主要结局
Effect of paricalcitol on proteinuria reduction
时间窗: 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.
次要结局
未报告次要终点
研究者
Eleonora Riccio
MD
Federico II University