JPRN-jRCT2071220110进行中（未招募）3 期
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
Hiroiku Furukawa0 个研究点目标入组 15 人开始时间: 2023年2月28日最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 15
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 至 ot applicable（—）
- 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)
研究者
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