Open-label Individual Patient Study of Cytomegalovirus (CMV)-Specific Donor-derived T Lymphocytes (DTL) for the Treatment of Recalcitrant CMV Infection in a Patient With Primary Immunodeficiency
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Feasibility of study
研究概览
简要总结
Treatment of CMV in a patient with profound combined immunodeficiency, who has viremia and pneumonia, using CMV-specific donor-derived T lymphocytes (CMV-VST).
详细描述
Treatment of CMV in a patient with profound combined immunodeficiency, who has viremia and pneumonia, using CMV-specific donor-derived T lymphocytes (CMV-VST), a cell therapy product containing a mixture of donor lymphocytes, reactive to peptides derived from cytomegalovirus.
After having receipt of therapy, the patient will have clinical assessments twice a week until discharge from the inpatient unit. After discharge, assessments will be performed on a weekly basis for three months. From 3-12 months, the patient will be seen monthly and then every three months till 2 years post planned hematopoietic stem cell transplantation. After 2 years, survival status will be assessed every 6 months through year 15.
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
- 0 Years 至 20 Years（Child, Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •profound combined immunodeficiency
- •cytomegalovirus (CMV) infection
- •pneumonia
排除标准
- •Receiving a steroid dose of ≥ 0.5 mg/kg of prednisolone equivalent
- •Receiving antithymocyte globulin or similar anti-T-cell antibody therapy, methotrexate, or other antimetabolite-type immunosuppressants that are toxic to proliferating T cells, and extracorporeal
- •Receiving checkpoint inhibitor agents (eg, nivolumab, pembrolizumab, ipilimumab) are within 3 drug half-lives of the most recent dose to cycle 1 day
- •Administration of another investigational product
研究组 & 干预措施
CMV-specific donor-derived T lymphocytes (CMV-VST).
Mixture of donor lymphocytes, reactive to peptides derived from cytomegalovirus.
干预措施: CMV-VST (Biological)
结局指标
主要结局
Feasibility of study
时间窗: Enrollment to 24 months
Evaluate the feasibility of conducting this study, evaluated in terms of whether or not the study could be completed as laid out in the protocol in the time allotted.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
时间窗: Weekly to 3 months
AEs assessed according to CTCAE grading criteria.
Efficacy of Intervention
时间窗: Every 3 months from 12-24 months
Change in viremia from baseline according to PCR testing after the intervention
次要结局
- Engraftment failure(Enrollment to 24 months)
- Graft versus host disease(Enrollment to 24 months)
- Transplant associated thrombotic microangiopathy(Enrollment to 24 months)
- Death(Enrollment to 24 months)
- CMV-reactive T cell number(Every 3 months from 12-24 months)
- CMV-reactive T cell phenotyping(Every 3 months from 12-24 months)
- RNA sequencing(Every 3 months from 12-24 months)
- Serum cytokine analysis(Every 3 months from 12-24 months)
- Total CMV-reactive T cell activity(Every 3 months from 12-24 months)