HAPLEUK17, Haploidentical Hematopoietic Cell Transplantation for Children With Hematologic Malignancies and Myelodysplasia
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 20
- 试验地点
- 2
- 主要终点
- Measure rates of immune reconstitution
研究概览
简要总结
This is a Phase I/II study designed to evaluate the kinetics of hematopoietic reconstitution and the incidence of acute chronic GVHD after partially matched related donor hematopoietic cell transplantation using an αβTCR/CD19+ cell depleted graft.
详细描述
Less than 30% of patients undergoing hematopoietic cell transplantation (HCT) will have an HLA-matched sibling donor. There is a high likelihood of being unable to identify a perfect HLA matched unrelated donor, and the time to procure the marrow if such a donor is available is generally >3 months. An emerging body of literature suggests that related haploidentical HCT with innovative graft engineering may provide equal, or possibly superior, outcomes to conventional unrelated donors. This protocol is designed to test the hypothesis that HCT using an αβT cell / CD19+ B cell depleted graft from partially matched related donors will result in rapid, durable hematopoietic engraftment and rapid immune reconstitution with an acceptably low risk of severe acute and chronic GVHD.
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
- — 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Patient lacks an HLA matched sibling donor.
- •Meets criteria nonhematopoietic organ function according to NCH BMT SOP
- •If subjects have received a first HCT, they must be eligible for a second HCT if their disease has recurred.
- •High resolution HLA and KIR typing
- •The subject cannot have an active untreated infection. Viremia by PCR analysis is not considered an active infection but may require immediate viral prophylaxis. Patients with possible fungal infections must have had at least 2 weeks of appropriate anti- fungal therapy and be asymptomatic.
- •Negative pregnancy test for females ≥11 years of age or post- menarche.
- •Sexually active males and females of childbearing potential must agree to use a form of contraception considered effective and medically acceptable by the Investigator. (Non-childbearing potential defined as pre-menarche, greater than one year post-menopausal or surgically sterilized).
- •Subjects must be ≤30 years at the time of consent.
- •Signed consent by parent/guardian and assent if appropriate for subjects < 18 years of age. Signed consent by patient/subject if ≥18 years of age.
排除标准
- •Patient does not have a suitable donor who is willing and able (meets donor criteria).
- •Patient has donor-specific anti-HLA antibodies at the time of enrollment
- •Patient reports a history of allergic reactions to murine protein
- •Donor Eligibility:
- •The donor must be ≥18 years of age at the time of the informed consent conference.
- •The donor must be a related donor
- •The donor will be evaluated according to the current NCH BMT SOP 04 and must meet all criteria.
- •The donor must be able and willing to undergo G-CSF mobilization and stem cell apheresis.
- •The patient does not have donor specific anti-HLA antibodies
结局指标
主要结局
Measure rates of immune reconstitution
时间窗: 4 years
Patients undergoing partially matched related donor hematopoietic cell transplantation with an αβT cell / CD19+ B cell depleted graft should have a improved rate of immune reconstitution.
Measure incidence of chronic GVHD
时间窗: 4 years
Patients undergoing partially matched related donor hematopoietic cell transplantation with an αβT cell / CD19+ B cell depleted graft should have a lower incidence of acute GVHD.
Measure rates of neutrophil and platelet engraftment
时间窗: 4 years
Patients undergoing partially matched related donor hematopoietic cell transplantation with an αβT cell / CD19+ B cell depleted graft should have an improved rate of engraftment.
Measure incidence of acute GVHD
时间窗: 4 years
Patients undergoing partially matched related donor hematopoietic cell transplantation with an αβT cell / CD19+ B cell depleted graft should have a lower incidence of acute and chronic GVHD.
Measure rates of platelet engraftment
时间窗: 4 years
Patients undergoing partially matched related donor hematopoietic cell transplantation with an αβT cell / CD19+ B cell depleted graft should have an improved rate of engraftment.
次要结局
- Measure overall survival(4 years)
- Measure relapse rate(4 years)
- Measure disease free survival(4 years)
- Define nonhematopoietic regimen related toxicities(4 years)