Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum (PXE)
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 主要终点
- Concentration area under the curve 0-t of pyrophosphate
研究概览
简要总结
Pyrophosphate is an endogenous, non-toxic metabolite inhibiting soft tissue calcification. The aim of our study is to find optimal dosing and safety of oral disodium-PPi (Na2H2PPi). Absorption curves (pharmacokinetics), AUC0-t, Cmax and Tmax for PPi and phosphate will be provided for healthy controls and PXE-patients both fasting and with standard meal intake.
详细描述
Phase II oral capsulized disodium-PPi (Na2H2PPi) powder absorption study in subjects with PXE (n=8-12) will be done in the ward of Internal Medicine of Tampere University Hospital and University Medical Center Utrecht.
After a 10-hour fast at 8.00 a.m. 30 mg/kg (first day) and 50 mg/kg (second day) capsulized dose of PPi will be given with 2 dl water. At 12.00 a.m. another capsulized 30 mg/kg (first day) or 50 mg/kg (second day) single dose of PPi with a standard mixed meal (lunch) will be given with 2 dl water at the time when a subject starts eating.
Plasma and urine sampling include plasma and spot urine electrolytes, creatinine, and pyrophosphate. Plasma sampling will be done at 0, 15, 30, 60, 120 and 240 min after ingestion of PPi. Urine spot sample will be taken at 0 and 240 min after ingestion of PPi.
Physical activity is restricted. Side-effects will be recorded.
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
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults (>18 yrs)
- •clinically and genetically proven PXE
- •Body mass index (BMI) 18,5-34,9 kg/m2
排除标准
- •Special groups according to researchers' decision.
- •Pregnancy
- •No effective contraception in females in child-bearing age.
研究组 & 干预措施
Open label
Disodiumpyrophosphate, capsuled powder, First day: 30 mg/kg fasting at 08.00 and with standard mixed meal at 12.00 Second day: 50 mg/kg fasting at 08.00 and with standard mixed meal at 12.00
干预措施: Disodium Pyrophosphate (Drug)
结局指标
主要结局
Concentration area under the curve 0-t of pyrophosphate
时间窗: two days
Maximal concentration of pyrophosphate
时间窗: two days
Cmax
Timepoint of maximal pyrophosphate concentration
时间窗: two days
Tmax
次要结局
- Concentration area under the curve 0-t of phosphate(two days)
- Maximal concentration of phosphate(two days)
- Timepoint of maximal phosphate concentration(two days)