An Observational Study Research to Collect the Effectiveness and Safety Data of KUVAN® Retrospectively in Chinese Subjects With Hyperphenylalaninemia (HPA) Caused by Tetrahydrobiopterin (BH4) Deficiency
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 26
- 试验地点
- 1
- 主要终点
- Number of participants with AE reported (% of patient with AE reported)
研究概览
简要总结
A retrospective study to collect the effectiveness and safety data of the past treatment with KUVAN in Chinese patients with HPA caused by BH4 deficiency. The data was collected from relevant past medical history and past clinical and safety assessments.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Willing and able to provide informed consent (unless exemption of obtaining consent is obtained as per local regulation). Assent (if required defined by local regulation and requirement) should also be obtained from the subject and the legal authorized representative.
- •Diagnosed with BH4 deficiency per local practice.
- •KUVAN® was taken at least 1 dose to treat HPA caused by BH4 deficiency during the period of observation.
- •Baseline Phe concentration ≥ 450 µmol/L
排除标准
- •Subject diagnosed to have Phenylketonuria (PKU)
- •Has any condition that, in the view of the Investigator, the medical record of the subject in the duration of observation is not reliable, or not accessible.
结局指标
主要结局
Number of participants with AE reported (% of patient with AE reported)
时间窗: 2010-2015
Retrospective body weight as indicated in medical note (kg)
时间窗: 2010-2015
Retrospective KUVAN treatment history - dose (mg/kg/day),
时间窗: 2010-2015
Retrospective KUVAN treatment history - length of exposure (days)
时间窗: 2010-2015
Retrospective Baseline Phe concentration ≥ 450 μmol/L - laboratory testing results (μmol/L)
时间窗: 2010-2015
Incidents and severity of AE reported (% of incident / % of each severity)
时间窗: 2010-2015
Retrospective intelligence development status via China local standard development assessment method (score)
时间窗: 2010-2015
Diagnosis on BH4 deficiency based on medical history - laboratory testing results indicating sub-type (% in each sub-type) • Incidents and severity of AE reported (% of incident / % of each severity)
时间窗: 2010-2015
Retrospective body height as indicated in medical note (cm)
时间窗: 2010-2015
Retrospective occipital / frontal circumferences as indicated in medical note (cm)
时间窗: 2010-2015
Retrospective blood Phe level as indicated in laboratory testing results (μmol/L)
时间窗: 2010-2015
次要结局
未报告次要终点