NCT02859727终止2 期
An Open-label, Non-randomized Extension Study to Evaluate the Long Term Safety, Tolerability, Efficacy and Pharmacokinetics of CDZ173 in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 37
- 试验地点
- 8
- 主要终点
- To Evaluate the Number of Participants With (S)AEs During Treatment With CDZ173
研究概览
简要总结
This study is designed to provide long-term CDZ173 treatment, a selective PI3Kδ inhibitor, to the patients with genetically activated PI3Kδ, i.e., patients with APDS/PASLI who participated in the CCDZ173X2201 study or who were treated previously with PI3Kδ inhibitors other than CDZ173. The study is open-label designed to establish the long-term safety, tolerability, efficay and pharmacokinetics of CDZ173 in the target population.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent must be obtained before any assessment is performed.
- •Paients must have participated in the study CCDZ173X2201 or were treated previously with PI3Kδ inhibitors other than CDZ
- •Patients who are deemed by the Investigator to benefit from PI3Kδ inhibitor therapy.
- •Patients or their legal representatives (for patients under the age of 18 years) must be able to communicate well with the Investigator, to understand and comply with the requirements of the study.
- •Documented APDS/PASLI-associated genetic PI3K delta mutation.
排除标准
- •- Any medically significant disease or condition that is unrelated to APDS/PASLI
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
CDZ173
Experimental
140mg/day
干预措施: CDZ173 (Drug)
结局指标
主要结局
To Evaluate the Number of Participants With (S)AEs During Treatment With CDZ173
时间窗: 6 years 3 months
Number of participants with adverse events reported, including serious adverse events
次要结局
- To Evaluate the Long Term Efficacy of CDZ173 Using SF-36 General Health Score(At baseline, after 1 year, after 3 years, after 4 years and after 5 years of study participation)
研究者
研究点 (8)
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