Use of Tolvaptan, a Vasopressin Antagonist, to Increase Urine Dilution and Reduce Cystine Urolithiasis Among Patients With Homozygous Cystinuria: a Pilot Investigation
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8)
研究概览
简要总结
The goal of this research project is to establish that in short-term use, tolvaptan is a safe and potentially effective new therapy for cystinuria, by conducting a short-term pilot study of the safety and tolerability of this drug, and assess impact on urinary stone risk parameters, among adolescent and young adult patients with clinical cystinuria.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 29 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females age 12 - 29 years
- •Weight ≥ 25kg (55 lbs)
- •Confirmed cystinuria diagnosis
- •Specific blood test levels (done within the past 6 months)
排除标准
- •Concurrent non-renal disease that might increase risk of complications due to aquaresis
- •Liver or biliary disease (chronic or acute)
- •Malabsorption syndrome or other gastrointestinal condition that may interfere with response to therapy
- •Non-cutaneous malignancy within last 5 years
- •History of adverse reaction or allergy to Tolvaptan or other arginine vasopressin V2-receptor antagonists
研究组 & 干预措施
Tolvaptan
干预措施: Tolvaptan (Drug)
结局指标
主要结局
Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8)
时间窗: 23 days
The primary outcome was urinary cystine supersaturation as measured by "cystine capacity". This proprietary test (Litholink Corp., Chicago IL) is reported as a value in mg/L above or below zero, with positive values indicating urine undersaturated with cystine, while negative values indicate that the urine is supersaturated with cystine. Four 24-hour urine samples were obtained during the study: one at baseline 3-6 days prior to the measurement, one on day 3-4 of the dosing period, one on day 7-8 of the dosing period, and one 3-6 days after the washout period. Each 24 hour urine sample was sent individually for analysis by Litholink Corp, which performs the cystine assays.
次要结局
- Urine Osmolality at High Dose (Day 8)(11 days)
研究者
Caleb Nelson
MD, MPH
Boston Children's Hospital