NCT02007863已完成不适用
Treatment Protocol: Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia Using Conditioning Regimen Without Radiation
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 2
- 试验地点
- 2
- 主要终点
- Number of Successful Unrelated Cord Blood (UCB) Transplants
研究概览
简要总结
Unrelated Cord Blood (UCB) transplant in children is a viable stem cell transplant modality for patients with leukemia and myelodysplasia. UCB is now considered "Standard Of Care" in cases where a suitable living bone marrow donor is not available. The survival of UCB is similar to Matched Unrelated Marrow Transplant. This study is considered "Research" since UCB is not a licensed product and requires investigational new drug (IND). THERE ARE NO SPECIFIC RESEARCH QUESTIONS IN THIS PROTOCOL. This protocol merely provides UCB as a stem cell treatment modality to pediatric patients who may require it after a conditioning regimen that excludes Total Body Irradiation.
详细描述
The preparative regimen will consist of:
- Fludarabine: 25 mg/m2/day IV x 5 doses on Days -13, to -9
- Busulfan 1mg/kg IV every 6 hrs x 16 doses on Days -8 to -5
- Melphalan 45 mg/m2/day IV x 3 doses on days -4 to -2
- ATGAM 30mg/kg/day x 3 doses on Days -3 to -1
- Day 0 will be the day of the UCB Transplant
- The graft-versus-host-disease (GVHD) prophylaxis will be Cyclosporin A to maintain level 200-400 beginning on Day -3, through 200-400. Solumedrol at 1mg/kg/day on Day 1 until D+4, then solumedrol 2mg/kg/day until Day +19 or till absolute neutrophil count (ANC) reaches 500/mm2, then taper by 0.2 mg/kg/week.
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 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must be up to 21 years of age
- •Patients cannot receive total body irradiation (TBI) because of:
- •Young age - < 2 years at diagnosis of leukemia resulting in an age < 4 years at transplantation (due to risk of severe growth retardation and brain damage).
- •Inability to tolerate TBI because of prior radiation or organ toxicity.
- •Refractory/multiply relapsed leukemia, for which a traditional TBI/cyclophosphamide regimen would unlikely lead to a successful outcome.
- •Patients must have a partially human leukocyte antigen (HLA)-matched UCB unit. Unit must be HLA-matched minimally at 4 of 6 HLA-A and B (at intermediate resolution by molecular typing) and DRB1 (at high resolution by molecular typing) loci with the patient. The unit must deliver a pre-cryopreserved nucleated cell dose of at least 2.5 x 10^7 per kilogram.
- •Acute myelogenous leukemia (AML) at the following stages:
- •High risk first complete remission (CR1), defined as:
- •Having preceding myelodysplasia (MDS)
- •High risk cytogenetics (High-risk cytogenetics: del (5q) -5, -7, abn (3q), t (6;9) complex karyotype (>= 5 abnormalities)
- •Requiring > 2 cycles chemotherapy to obtain CR;
- •Second or greater CR.
- •First relapse with < 25% blasts in bone marrow.
- •Patients with therapy-related AML whose prior malignancy has been in remission for at least 12 months.
- •Acute lymphocytic leukemia (ALL) at the following stages:
- •High risk first remission, defined as:
- •Ph+ ALL; or,
- •Myeloid/lymphoid leukemia (MLL) rearrangement with slow early response [defined as having M2 (5-25% blasts) or M3 (>25% blasts on bone marrow examination on Day 14 of induction therapy)]; or,
- •Hypodiploidy (< 44 chromosomes or DNA index < 0.81); or,
- •End of induction M3 bone marrow; or,
- •End of induction M2 with M2-3 at Day
- •High risk second remission, defined as:
- •Bone marrow relapse < 36 months from induction; or,
- •T-lineage relapse at any time; or,
- •Very early isolated central nervous system (CNS) relapse (6 months from diagnosis); or,
- •Slow reinduction (M2-3 at Day 28) after relapse at any time.
- •Any third or subsequent CR.
- •Biphenotypic or undifferentiated leukemia in any CR or if in 1st relapse must have < 25% blasts in bone marrow (BM).
- •MDS at any stage.
- •Chronic myelogenous leukemia (CML) in chronic or accelerated phase.
- •All patients with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for study.
- •Patients ≥ 16 years old must have a Karnofsky score ≥ 70% and patients < 16 years old must have a Lansky score ≥ 70%.
- •Signed informed consent.
- •Patients with adequate physical function as measured by:
- •Cardiac: Left ventricular ejection fraction at rest must be > 40%, or shortening fraction > 26%
- •Hepatic: Bilirubin ≤ 2.5 mg/dL; and alanine transaminase (ALT), aspartate transaminase (AST) and Alkaline Phosphatase ≤ 5 x upper limit of normal (ULN)
- •Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) > 70 mL/min/1.73 m
- •Pulmonary: Diffusing capacity of the lungs for carbon monoxide (DLCO), Forced expiratory volume in 1 second (FEV1), Forced vital capacity (FVC) (diffusion capacity) > 50% of predicted (corrected for hemoglobin); if unable to perform pulmonary function tests, then O2 saturation > 92% of room air.
排除标准
- •Pregnant (B-positive HCG) or breastfeeding.
- •Evidence of HIV infection or HIV positive serology.
- •Current uncontrolled bacterial, viral or fungal infection (currently taking medication and progression of clinical symptoms).
- •Autologous transplant < 6 months prior to enrollment.
- •Prior autologous transplant for the disease for which the UCB transplant will be performed.
- •Allogeneic hematopoietic stem cell transplant < 6 months prior to enrollment.
- •Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment
- •Active CNS leukemia.
- •Requirement of supplemental oxygen.
- •HLA-matched related donor able to donate.
研究组 & 干预措施
Umbilical Cord Blood + Chemotherapy
Experimental
Umbilical Cord Blood transfusion + Chemotherapy (Fludarabine + Busulfan + Melphalan)
干预措施: Umbilical Cord Blood Transfusion (Procedure)
Umbilical Cord Blood + Chemotherapy
Experimental
Umbilical Cord Blood transfusion + Chemotherapy (Fludarabine + Busulfan + Melphalan)
干预措施: Fludarabine (Drug)
Umbilical Cord Blood + Chemotherapy
Experimental
Umbilical Cord Blood transfusion + Chemotherapy (Fludarabine + Busulfan + Melphalan)
干预措施: Busulfan (Drug)
Umbilical Cord Blood + Chemotherapy
Experimental
Umbilical Cord Blood transfusion + Chemotherapy (Fludarabine + Busulfan + Melphalan)
干预措施: Melphalan (Drug)
结局指标
主要结局
Number of Successful Unrelated Cord Blood (UCB) Transplants
时间窗: 2 Years
The number of patients who received successful UCB transplants as evidenced by absolute neutrophil recovery.
次要结局
未报告次要终点
研究者
Martin Andreansky
Associate Professor of Clinical
University of Miami
研究点 (2)
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