An Open-label, Multi-center Phase I/II Study to Assess the Safety and the Efficacy of SMART101 After Haploidentical Peripheral Blood Stem Transplantation With Post-transplant Cyclophosphamide in Subjects With Hematological Malignancies
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 4
- 主要终点
- Occurrence of Unexpected Unacceptable Toxicities (UUT) following the administration of SMART101.
研究概览
简要总结
The purpose of this study is to evaluate the safety and the efficacy of SMART101 (Human T Lymphoid Progenitors (HTLP)) injection to accelerate immune reconstitution after haploidentical hematopoietic stem cell transplantation (HSCT) with post-transplant cyclophosphamide (PT-Cy) in adult patients with hematological malignancies.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
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
- •Main Inclusion Criteria:
- •Patients with AML, ALL or MDS eligible for an allogeneic HSCT with a haploidentical donor with post-transplant cyclophosphamide.
- •Patients must be ≥ 18 years of age at the time of signing the ICF.
- •Patients must have a Karnofsky index ≥ 70%.
- •Patients must have a left ventricular ejection fraction of ≥40%.
- •Patients must have an intact pulmonary function or Diffusing capacity of the Lungs for Carbon Monoxide (DLCO) ≥ 45% of predicted.
- •Patients must have adequate hepatic and renal functions, as assessed by standard laboratory criteria.
排除标准
- •Patients who have received prior allogeneic stem cell transplantation.
- •Patients who have received prior treatment with another cellular therapy within 4 weeks before the planned day of SMART101 infusion.
- •Patients who plan to receive, are concurrently receiving or have received any investigational agent within 4 weeks before the planned day of SMART101 infusion.
研究组 & 干预措施
Patients with acute leukemia or myelodysplastic syndrome and eligible for an haplo PT-Cy HSCT
Segment 1: 3 dose-level SMART101 cells/infusion
- 1.5 x 106 CD7+ cells per kg of body weight
- 4.5 x 106 CD7+ cells per kg of body weight
- 9.0 x 106 CD7+ cells per kg of body weight
Segment 2:
2 cohorts of patients will be included in the study based on the type of conditioning regimen:
- The cohort A will include up to 17 patients receiving a myeloablative conditioning (MAC).
- The cohort B will include up to 17 patients receiving a reduced intensity conditioning (RIC).
- Enrollment of patients in each cohort will be done in parallel.
干预措施: Allogeneic T cell progenitors, cultured ex-vivo (Biological)
结局指标
主要结局
Occurrence of Unexpected Unacceptable Toxicities (UUT) following the administration of SMART101.
时间窗: 14 days post SMART101 infusion
To evaluate the safety of SMART101.
CD4+ T cell count.
时间窗: 100 days post-HSCT
to evaluate the efficacy of the study drug
次要结局
- Occurrence of adverse events (AEs)(up to 24 months post-HSCT)
- Cumulative incidence of infections(Day 100, and Months 6 and 12 post-HSCT)
- T cell immune reconstitution(up to 12 months post-HSCT)
- Non-relapse mortality (NRM)(Day 100, and Months 6, 12 and 24 post-HSCT)