TCTR20231002005尚未招募不适用
Evaluation of responsiveness to Sapropterin treatment in Thai patient with phenylketonuria
适应症
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 8
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 1 Hours 至 18 Years（—）
- 性别
- All
入选标准
- •Thai patient with PKU who follow up in Faculty of Medicine Siriraj Hospital.
- •The neonates who are positive from newborn screening and suspected to be PKU.
排除标准
- •The patient's serum Phenylalanine level is lower than 360 umol/L or higher than 2,000 umol/L
- •The PAH gene mutation is not identified in the patient.
- •The patient deny the treatment by Sapropterin.
- •The patient is older than 18 years.
研究者
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