NCT01281878已完成2 期
PILOTSTUDIE ZUR PYRIDOXALPHOSPHATTHERAPIE BEI PATIENTEN MIT PRIMÄRER HYPEROXALURIE TYP I (PHOX-B6-PILOT) Pilot Trial on Treatment of Patients With Primary Hyperoxaluria Type I With Pyridoxal-phosphate
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- The primary endpoint of the study is the reduction of the urinary oxalate excretion (percentage change in urinary oxalate, expressed as mmol/1.73 m2 /day) at week 24 compared to baseline.
研究概览
简要总结
In this study the investigators will prospectively analyze the reduction of urinary oxalate excretion under the treatment with PLP in dosages of 5mg/kg/day up to 20 mg/kg/day and serum level response relationship with PLP as an i.v. solution used orally in 12 patients with primary hyperoxaluria type I as an inherited autosomal-recessive-disorder leading to increased endogenous oxalate production, urolithiasis and end stage renal disease.
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
- 5 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documentation of diagnosis of PH I by any one of the following:
- •Liver biopsy confirmation of deficient liver specific peroxisomal alanine-glyoxylate aminotransferase, (AGT or mislocalization of AGT from peroxisomes to mitochondria)
- •Homozygosity or compound heterozygosity for a known mutation in the causative gene (AGXT) for PH I
- •Male or female subjects between 5 years and 60 years of age
- •Renal function defined as an estimated GFR > 60 ml/min normalized to 1.73 m2 body surface area
- •Subjects receiving pyridoxal-phosphate before the study must be willing to discontinue therapy with pyridoxal-phosphate for a wash out phase of at least 4 weeks but always until normalization of serum pyridoxal-phosphate levels
- •Written informed consent from patients and/or legally acceptable representatives
排除标准
- •Pregnant or lactating women
- •Women of child-bearing potential who are not using a highly effective contraception method with a pearl-index <
- •Highly effective contraception methods are oral, transdermal, injectable, or implanted contraceptives, IUD, abstinence, or sterile sexual partner and must agree to continue using such precautions during the pyridoxal-phosphate study
- •Subjects post liver or kidney transplantation or combined transplantation
- •Chronic diarrhoea with the risk of malabsorption
- •Any other abnormal finding such as physical examination or laboratory evaluation, in the opinion of the investigator, is indicative of a disease that would compromise the safety taking pyridoxal-phosphate per os and the absorption
- •Subjects participating in other clinical trials with investigational products 4 weeks prior to trial entry, during the trial and 4 weeks after the trial
- •Subjects who are unable to take the trial medication
- •Subjects who are unable to collect 24-hour urine samples or follow other study procedures
- •Subjects who are under treatment with L-Dopa, Isoniazid, D-Penicillamine (interactions between these drugs and pyridoxal-phosphate are known and might influence serum pyridoxal-phosphate levels)
- •Subjects with known allergies to substances of contents (e.g. Potassium sorbet, raspberry syrup)
- •Subjects confined to an institution on judicial or official behalf
- •Subjects who are in dependency to the sponsor or the PI of the trial
研究组 & 干预措施
Pyridoxal-phosphate
Experimental
Treatment with pyridoxal-phosphate in increasing dosages every six weeks starting with 5mg/kg body weight up to 20 mg/kg body weight. treatment duration 24 weeks
干预措施: Vitamin B 6 (Drug)
结局指标
主要结局
The primary endpoint of the study is the reduction of the urinary oxalate excretion (percentage change in urinary oxalate, expressed as mmol/1.73 m2 /day) at week 24 compared to baseline.
时间窗: 6 month
次要结局
未报告次要终点
研究者
Prof. Dr. B. Hoppe
Prof. Dr. med. Bernd Hoppe
University of Cologne
研究点 (1)
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