jRCT2071220110进行中（未招募）不适用
An open-label, multi-center, switch-over study of the efficacy, safety and PK of HPN-100 compared to sodium phenyl butyrate in Japanese patients with urea cycle disorder, with a long-term safety extension
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 15
- 主要终点
- blood ammonia levels 24-hour area under the curve
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Single Arm Study
- 干预模型
- Single Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Open(masking Not Used)
入排标准
- 年龄范围
- 0age over 至 No limit（—）
- 性别
- All
入选标准
- •Patients diagnosed with urea cycle disorder (UCD) by genetic testing, enzymatic testing, and/or biochemical testing.
- •If participating in the switch-over part, patients being treated for UCD with NaPBA and who has not changed dosage and administration of NaPBA for at least one week prior to the start of study drug administration (Day 1).
- •If newly participating in the extension part, NaPBA has not been administered for at least 30 days prior to the start of study drug administration (Month 0).
- •Patients who are judged to be sufficiently capable of undergoing the tests and observations conducted in this study.
排除标准
- •Patients who have a blood ammonia level 100 micro mol/L (170 micro g/dL) or more at screening or has symptoms and/or signs of hyperammonemia within at least 2 weeks prior to the screening visit. If ammonia level is controlled and stable for at least 14 days, re-screening may be allowed at the discretion of the Investigator etc.
- •Patients with active infections (viral and bacterial) or other diseases that may affect blood ammonia levels.
- •Patients who have received sodium benzoate within 1 week prior to the start of study drug administration.
- •Patients with history of hypersensitivity to phenylbutyrate and/or phenylacetic acid.
- •Patients who have undergone liver transplantation (including hepatocellular transplantation)
结局指标
主要结局
blood ammonia levels 24-hour area under the curve
时间窗: Day 7 and Day 14
The blood ammonia levels 24-hour area under the curve at the last day of NaPBA administration (Day 7) and the last day of HPN-100 administration (Day 14)
次要结局
- Maximum blood ammonia levels(last day of NaPBA and HPN-100 administration)
- Mean blood ammonia levels(last day of NaPBA and HPN-100 administration)
- Patients with ammonia levels exceeding ULN(NaPBA and HPN-100 administration period)
- PK parameters of metabolites
- Cumulative 24-hour urinary PAGN excretion(last day of NaPBA and HPN-100 administration)
- Correlation between U-PAGN0-24 and AUCNH3
- Glutamine values(last day of NaPBA and HPN-100 administration)
- Adverse events
研究者
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