Clinical Study on the Safety and Efficacy of CD7 CAR-T Cell Sequential Allogeneic Hematopoietic Stem Cell Transplantation and Kidney Transplantation in the Treatment of Schimke Immuno-osseous Dysplasia
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs)
研究概览
简要总结
A Clinical Study on the Safety and Effectiveness of CD7 CAR-T Cell Sequential Allo-HSCT and Kidney Transplantation in the treatment of Schimke immuno-osseous dysplasia
详细描述
This is a single-arm, open-label, dose-escalation clinical trial to evaluate the safety and efficacy of CD7 CAR-T Cell Sequential Allo-HSCT and Kidney Transplantation in patients with Schimke immuno-osseous dysplasia. It is planned to enroll 20 participants in this trial.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed as SIOD and was in stage 5 of chronic kidney disease
- •Having allogeneic HSCT indications, at least suitable donors (relatives) for haploidentical allogeneic transplantation and kidneys from stem cell transplantation donors;
- •serum total bilirubin ≤ 1.5 times the upper limit of normal, and serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were both ≤ 3 times the upper limit of the normal range.
- •Echocardiogram shows left ventricular ejection fraction (LVEF) ≥ 50%;
- •There is no active pulmonary infection, and the oxygen saturation during air inhalation is more than 92%；
- •Estimated survival time ≥ 3 months;
- •ECOG performance status 0 to 1;
- •Pregnant/lactating women, or male or female patients who have fertility and are willing to take effective contraceptive measures at least 6 months after the last cell infusion during the study period;
- •Those who voluntarily participated in this trial and provided informed consent;
排除标准
- •Allergic to pretreatment measures
- •received any containing ATG/ALG such IST、alemtuzumab、high-dose cyclophosphamide (≥ 45mg/kg/day) , received CsA treatment within 6 months, or used thrombopoietin receptor (tpo-r) agonists in the past;
- •Patients with the history of epilepsy or other CNS disease;
- •Patients with prolonged QT interval time or severe heart disease;
- •Previous recipients of allogeneic hematopoietic stem cell transplantation or organ transplantation
- •People infected with HIV, active hepatitis B or hepatitis C virus, and patients with active infection who are not cured;
- •The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
- •Patients with malignant tumor;
- •People with other genetic diseases；
- •After receiving CD7 car-t treatment, patients who were unable to accept subsequent kidney transplantation due to severe infection or poor amplification of car-t in vivo.
- •Any situation that researchers believe may increase the risk to the subjects or interfere with the trial results.
研究组 & 干预措施
Treatment Group
Schimke Immuno-osseous Dysplasia
干预措施: CD7 CAR-T cells injection (Biological)
Treatment Group
Schimke Immuno-osseous Dysplasia
干预措施: Allo-HSCT (Procedure)
Treatment Group
Schimke Immuno-osseous Dysplasia
干预措施: Kidney Transplantation (Procedure)
结局指标
主要结局
Incidence of treatment-emergent adverse events (TEAEs)
时间窗: Up to 2 years after Treatment
Incidence of treatment-emergent adverse events
Transplant related mortality rate
时间窗: Up to 100 days after Treatment
The proportion of patients who died after transplantation to the total number of transplant patients during the same period
次要结局
- Allogeneic hematopoietic stem cell transplant implantation rate(Up to 100 days after Treatment)
- Kidney transplantation implantation rate(Up to 100 days after Treatment)
- Time to neutrophil and platelet engraftment(Up to 30 days after Treatment)
- Disease-feesurvival，DFS(Up to 2 years after Treatment)
- Overall survival, OS(Up to 2 years after Treatment)
研究者
He Huang
Principal Investigator
First Affiliated Hospital of Zhejiang University