Cerliponase Alfa Observational Study
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 35
- 试验地点
- 16
- 主要终点
- Safety surveillance of cerliponase alfa
研究概览
简要总结
This is a multicenter, observational study for patients with a confirmed diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who intend to be or are currently being treated with cerliponase alfa. Patients receiving or expected to receive cerliponase alfa within 60 days of signing the informed consent form (ICF) may be eligible to enroll in the study, assuming all regulatory requirements for sites that have agreed to participate and protocol inclusion criteria are met. Data may be collected for all or some of the assessments as outlined in the protocol, dependent upon the clinic's and/or individual patient's standard of care.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with CLN2 disease.
- •Currently receiving or plan to begin treatment with cerliponase alfa.
- •Written informed consent/assent obtained.
排除标准
- •1. Currently receiving treatment in another investigational device or drug study.
研究组 & 干预措施
Cerliponase alfa patients
Patients who are currently on or plan to start taking cerliponase alfa within 60 days of signing the study informed consent form.
干预措施: Cerliponase Alfa (Drug)
Cerliponase alfa patients
Patients who are currently on or plan to start taking cerliponase alfa within 60 days of signing the study informed consent form.
干预措施: Administration Kit (Device)
结局指标
主要结局
Safety surveillance of cerliponase alfa
时间窗: 10 years
To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).
次要结局
- Severe SAE impact on patient's motor and language functions(10 years)
- Hypersensitivity(10 years)