NCT06003881Approved For Marketing不适用
An Open-Label, Expanded Access Program of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- Approved For Marketing
研究概览
简要总结
The program is to provide access to an experimental drug called pozelimab and to document the long-term safety of pozelimab in patients with Protein-Losing Enteropathy (PLE). CD55-deficient PLE/CHAPLE disease is a rare inherited disease of the immune system. This can be a life-threatening condition that is usually found when patients are children.
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of CD55-deficient PLE/CHAPLE disease (based on a history of PLE), confirmed by biallelic CD55 loss-of-function mutation detected by genotype analysis (frameshift, nonsense mutations) as defined in the protocol
- •Written informed consent from parent/guardian for minor patients
- •Written assent from minor patients as appropriate (eg, above the age of 6 years or the applicable age per local regulatory requirements)
排除标准
- •Patients who discontinued the prior pozelimab study due to safety or lack of efficacy
- •Considered by the treating physician as inappropriate for this program for any reason as defined in the protocol
- •NOTE: Other protocol defined inclusion / exclusion criteria apply
研究者
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