A Phase 1 Trial of a Single ProHema® CB Product (Ex Vivo Modulated Human Cord Blood Cells) Following Myeloablative Conditioning for Pediatric Patients With Hematologic Malignancies
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 4
- 主要终点
- Safety Profile, Primarily Assessed by Neutrophil Engraftment
研究概览
简要总结
This is an open-label, safety study of a single ProHema-CB product administered following myeloablative conditioning regimen in pediatric subjects with hematologic malignancies.
详细描述
A maximum of 18 eligible male and female subjects (1 to 18 years old, inclusive) will be enrolled and treated in the trial at approximately 3 to 5 centers within the U.S. These 18 subjects will consist of 3 cohorts of 6 subjects each. The cohorts will be defined by age: 1 to 4 years; > 4 to 12 years; and > 12 to 18 years. These cohorts will be enrolled simultaneously.
All subjects will be admitted to the hospital, per institutional practice, and will receive a myeloablative conditioning regimen, after which they will receive an HLA-matched or partially matched ProHema-CB unit on study Day 0.
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
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects aged 1 to 18 years, inclusive.
- •Subjects with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate.
- •Acute Myelogenous Leukemia (AML) in high risk 1st or subsequent CR
- •Acute Lymphoblastic Leukemia (ALL) in CR
- •NK cell lymphoblastic leukemia in any CR
- •Biphenotypic or undifferentiated leukemia in 1st or subsequent CR
- •Myelodysplastic Syndrome (MDS) at any stage.
- •Chronic Myelogenous Leukemia (CML) All subjects with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for trial.
- •Lack of 5-6/6 HLA matched related or 8/8 HLA A, B, C, DRß1 matched unrelated donor; or unrelated donor not available within appropriate timeframe, as determined by the transplant physician.
- •Availability of suitable primary and secondary umbilical cord blood (UCB) units.
- •Adequate performance status, defined as:
- •Subjects ≥ 16 years: Karnofsky score ≥ 70%.
- •Subjects < 16 years: Lansky score ≥ 70%.
- •Cardiac: Left ventricular ejection fraction at rest must be > 40%, or shortening fraction > 26%.
- •Subjects > 10 years: DLCO (diffusion capacity) > 50% of predicted (corrected for hemoglobin)
- •FEV1, FVC > 50% of predicted; Note: If unable to perform pulmonary tests, then O2 saturation > 92% on room air.
- •Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) > 70mL/min/1.73m
- •Hepatic: Bilirubin ≤ 2.5 mg/dL (except in the case of Gilbert's syndrome or ongoing hemolytic anemia); and ALT, AST and Alkaline Phosphatase ≤ 5 × ULN.
- •Signed IRB approved Informed Consent Form (ICF).
排除标准
- •Female subjects that are pregnant or breastfeeding.
- •Evidence of HIV infection or HIV positive serology.
- •Current uncontrolled bacterial, viral or fungal infection.
- •Prior allogeneic hematopoietic stem cell transplant.
- •Autologous transplant < 12 months prior to enrollment.
- •Prior autologous transplant for the disease for which the UCB transplant is being performed.
- •Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment.
- •Inability to receive TBI.
- •Requirement of supplemental oxygen.
- •HLA-matched related donor able to donate.
- •Use of an investigational drug within 30 days prior to screening.
- •Subject is unlikely to comply with the protocol requirements, instructions and study-related restrictions
研究组 & 干预措施
ProHema-CB
All subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant.
ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight:
For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min.
For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour.
干预措施: Biological: ProHema-CB (Biological)
结局指标
主要结局
Safety Profile, Primarily Assessed by Neutrophil Engraftment
时间窗: Neutrophil engraftment by Day 42
To describe the safety profile of ProHema-CB after myeloablative conditioning in pediatric patients with hematologic malignancies. The safety profile will primarily be assessed by neutrophil engraftment.
次要结局
未报告次要终点