NCT04452513已完成不适用
A Prospective Clinical Study of Phenylketonuria (PKU)
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 32
- 试验地点
- 14
- 主要终点
- Change in Plasma Phe Levels
研究概览
简要总结
This is a study for adults and children ≥ 14 years old who have Phenylketonuria (PKU) with uncontrolled plasma Phe levels. No clinical intervention or study drug is provided by BioMarin in this study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency
- •Ability and willingness to maintain dietary protein intake consistent with baseline intake
- •Willingness and capable per investigator opinion to comply with study procedures and requirements
- •Plasma Phe Levels > 600umol/L
排除标准
- •Clinically significant liver dysfunction or disease
- •Prior treatment with gene therapy
- •Any condition that, in the opinion of the investigator or Sponsor, would prevent the subject from fully complying with the requirements of the study
结局指标
主要结局
Change in Plasma Phe Levels
时间窗: through study completion, an average of 2 years
次要结局
- Change in dietary protein intake from medical and intact food(through study completion, an average of 2 years)
研究者
研究点 (14)
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