Umbilical Cord Blood Transplant for Children With Lymphoid Hematological Malignancies (UCALL)
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 主要终点
- Overall Survival at 1 year after UCB transplant in pediatric patients
研究概览
简要总结
The purpose of this study is to determine the safety and effectiveness of UCBT to treat patients with lymphoid hematological diseases and to see if this treatment can decrease the incidence of leukemia relapse, GVHD and infections.
These patients have a type of blood cell disorder that is very hard to cure. This treatment that is being used in this trial is known as a stem cell transplant. This treatment might help the patient live longer without the disease. It uses much stronger doses of drugs and radiation to kill the diseased cells that could be given without the transplant. We also think that the healthy cells from the donor may help fight any diseased cells left after the transplant.
For the transplant to take place, we will administer stem cells from a 'donor' whose cells best 'match' the patient's. In this study umbilical cords will be the source of the stem cells. Before the transplant, two very strong drugs plus total body irradiation will be given to as preconditioning. This treatment will kill most of your blood-forming cells in the bone marrow. The patient will then get then healthy stem cells.
If the patient has the disease in the central nervous system (CNS), they will receive radiation to the head and spine before starting the conditioning. This is to try to get disease control in the CNS. Radiation will not be given for children under 2 yrs old.
Currently, many umbilical cord blood units are available in public banks for transplantation in patients lacking bone marrow donors. UCB transplants (UCBT) may offer several advantages over adult bone marrow or peripheral blood stem cell transplants, including:
- rapid availability,
- absence of donor risk,
- low risk of transmissible infectious diseases,
- low risk of acute GvHD (Graft vs. Host Disease)
The three main causes of death after umbilical cord blood transplantation for these kind of disorders are graft failure, infection and disease relapse.
In this study we are trying to address these three problems:
To help improve engraftment we will add the drug Fludarabine to Cytoxan and total body irradiation. Fludarabine is a very strong medicine. We will try to decrease infections and reduce leukemia relapse by using fludarabine instead of antithymocyte globulin (ATG).
详细描述
After the eligible criterion for treatment has been met and a suitable UCB stem donor has been found, the patient will have a central line placed.
Research Therapy:
After placement of the central line, the following chemotherapy will be given to after admission to the hospital and before the infusion of the umbilical cord blood stem cells:
- 9 - 6 days before the infusion: Total Body Irradiation (TBI) in two fractions ("doses") per days.
- 5 - 2 days before the infusion: Cytoxan given daily for 4 days, over 1 hour as an intravenous infusion. Mesna will be given per standards. Mesna is a drug given to decrease the side effects of Cytoxan. It will be given daily as an intravenous infusion while the patient receives the Cytoxan.
- 4 - 2 days before the infusion: Fludarabine given daily for 3 days over 1 hour as an intravenous infusion.
Stem cell transplant (intravenous infusion of the UCB stem cells) - defined as day 0 of the treatment. All other "numbered" days relate to this infusion date. For example, Day 1 is the first day after the stem cell transplant.
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Umbilical Cord Blood Transplant Treatment Plan
Cytoxan, Fludarabine, Total Body Irradiation (TBI), Cord Blood Stem Cell Infusion
干预措施: Cytoxan (Drug)
Umbilical Cord Blood Transplant Treatment Plan
Cytoxan, Fludarabine, Total Body Irradiation (TBI), Cord Blood Stem Cell Infusion
干预措施: Fludarabine (Drug)
Umbilical Cord Blood Transplant Treatment Plan
Cytoxan, Fludarabine, Total Body Irradiation (TBI), Cord Blood Stem Cell Infusion
干预措施: Total Body Irradiation (TBI) (Radiation)
Umbilical Cord Blood Transplant Treatment Plan
Cytoxan, Fludarabine, Total Body Irradiation (TBI), Cord Blood Stem Cell Infusion
干预措施: Cord Blood Stem Cell Infusion (Procedure)
结局指标
主要结局
Overall Survival at 1 year after UCB transplant in pediatric patients
时间窗: 1 year
To determine the survival rate at 1 year after umbilical cord blood transplant in pediatric patients with lymphoid hematological malignancies
次要结局
- Assessment of Platelet Count Recovery(Day 42)
- Number of participants with severe acute GVHD grade III-IV as an assessment of safety(Day 100)
- Number of participants with chronic GvHD as an assessment of safety(1 year)
- Assess relapse rate after transplant(1 and 3 years)
- Assessment of neutrophil count recovery(Day 42)
- To evaluate cell recovery and function(day 100, 6, 12, and 24 months)
- Assess leukemia free survival(1 and 3 years)
研究者
Caridad Martinez
Assistant Professor, Pediatric Hematology/Oncology, Center for Cell and Gene Therapy
Baylor College of Medicine