Kuvan® Adult Maternal Pediatric European Registry
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 627
- 试验地点
- 5
- 主要终点
- Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs)
研究概览
简要总结
Kuvan® is a synthetic copy of a body's own substance called tetrahydrobiopterin (BH4). BH4 is required by the body to use an amino acid called phenylalanine in order to build another substance called tyrosine.
Kuvan® received marketed authorisation in Europe in December 2008 and is now available in several European countries for the treatment of Hyperphenylalaninemia (HPA).
The primary objective is to assess the long-term safety in subjects treated with Kuvan®.
Secondary objectives are to provide additional information regarding:
- Safety in specific subject groups (elderly, pediatric, pregnant women and subjects with renal or hepatic insufficiency).
- Growth and neurocognitive outcomes for subjects with hyperphenylalaninemia (HPA) who are receiving treatment with Kuvan®.
- Progress and outcome of pregnancy for women with HPA who become pregnant while receiving treatment with Kuvan® (these women will be enrolled in a dedicated sub-registry).
- Assessment of adherence to diet and to Kuvan®.
- Assessment of long-term sensitivity to Kuvan®treatment.
详细描述
This is an observational, multicenter, drug registry Study. The study will have a total duration of 15 years, including a 10-year inclusion period. No diagnostic, therapeutic or experimental intervention is involved. Subjects will receive clinical assessments, medications and treatments solely as determined by their study physician.
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
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult or pediatric subject (no age limit) of either gender with HPA due to PKU or BH4 deficiency.
- •Have been shown to be responsive to BH4 or Kuvan. (Note: For Spain only-Have been shown to be responsive to BH4 or for the newly diagnosed subjects to be responsive to Kuvan as defined in the Summary of Product Characteristics [SmPC]).
- •Currently being treated with Kuvan® at a participating centre.
- •Subject or parent/legal guardian willing and able to provide written signed informed consent and given before any data collection. If a child is old enough to read and write, a separate assent form will be given.
排除标准
- •Known hypersensitivity to Kuvan®
- •Legal incapacity or limited legal capacity without legal guardian representation
- •Breast-feeding
结局指标
主要结局
Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs)
时间窗: A maximum of 15 years treatment duration.
次要结局
- Long-term sensitivity to Kuvan® treatment(A maximum of 15 years treatment duration.)
- Description on somatic growth (in BH4 deficient children < 3 years)(A maximum of 15 years treatment duration.)
- Neurological and psychiatric assessment(A maximum of 15 years treatment duration.)
- Blood Phe levels(A maximum of 15 years treatment duration.)
- Incidence of AEs/SAEs in specific population (elderly, children, subjects with renal or hepatic insufficiency)(A maximum of 15 years treatment duration.)
- Neurocognitive outcomes(A maximum of 15 years treatment duration.)
- Diet and Kuvan® treatment adherence(A maximum of 15 years treatment duration.)
- Tyrosine (Tyr) levels(A maximum of 15 years treatment duration.)
- Pregnancy and delivery outcomes(A maximum of 15 years treatment duration.)