NL-OMON25048尚未招募不适用
BH4 treatment in Phenylketonuria - comparing different practices of dosing
适应症
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 28
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
入选标准
- •In order to be eligible to participate in this study, a subject must meet all of the following criteria:
- •1. Diagnosed with phenylketonuria by newborn screening.
- •2. Diagnosed as a BH4-responder after a 48-h BH4 loading test and a treatment trial
- •3. Currently uses sapropterin as part of treatment (with a minimum of three months)
- •4. Age = 4 years
- •5. Good metabolic control for the previous 6 months, meaning 75% of the Phe levels are within therapeutic target levels with a minimum of four values.
排除标准
- •A potential subject who meets any of the following criteria will be excluded from participation in this study:
- •Pregnancy, planning pregnancy or lactation
研究者
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