ASCEND-JC: A Multi-center, Randomized, Double-blind, Phase 2 Study, Evaluating JCPyV-specific T Cell Therapy for the Treatment of PML
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 60
- 主要终点
- To evaluate the effect of CE-VST01-JC on time to disease progression, as measured by mRS (modified Rankin Score)
研究概览
简要总结
This is a randomized, double-blinded, Phase 2 trial in patients with PML due to JCPyV. Patients will receive treatment with a matched virus-specific T-cell product (CE VST01-JC) or placebo, and will then be monitored for response to therapy.
详细描述
Progressive multifocal leukoencephalopathy (PML) is a demyelinating disease of the central nervous system caused by JC polyomavirus (JCPyV) occurring in immunocompromised patients who face a disease that is usually progressive and often fatal (Padgett 1971; Tan 2010). Annually, it is estimated that 4,000 people develop PML in the United States and Europe combined (NORD 2015).
This is a randomized, double- blinded, Phase 2 trial in patients with PML due to JCPyV. Patients will receive treatment with a matched virus-specific T-cell product (CE VST01-JC) or placebo, and will then be monitored for response to therapy. The study is designed to evaluate whether CE-VST01-JC infusions will slow and ultimately halt neurological progression in patients with PML compared with placebo as evaluated by modified Rankin Score (mRS).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects aged 18 years or older, with actively progressing PML confirmed by MRI and JCPyV present in CSF or in brain biopsy tissue (diagnosis confirmed according to algorithm diagnostic criteria for PML developed by the American Academy of Neurology).
排除标准
- 未提供
研究组 & 干预措施
CE-VST01-JC
干预措施: CE-VST01-JC (Biological)
Placebo
干预措施: CE-VST01-JC (Biological)
结局指标
主要结局
To evaluate the effect of CE-VST01-JC on time to disease progression, as measured by mRS (modified Rankin Score)
时间窗: 1 year
次要结局
未报告次要终点