Umbilical Cord Blood Transplant for Congenital Pediatric Disorders
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Overall Survival at 1 Year After Umbilical Cord Blood 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 Umbilical Cord Blood Transplant (UCBT) to treat the patient's disease, and to see if this treatment can decrease the incidence of GVHD.
This study is for patients that were born with a disease that affects their body's metabolism or immune system. The doctor plans to treat the patient for this illness with a stem cell transplant.
While improved medical care has allowed many people with these diseases to live longer, the only way to truly cure the diseases is by means of a stem cell transplant from a donor who does not have the disease. A stem cell transplant will replace sick cells with new healthy donor cells. Stem cells grow into different types of blood cells that people need, including red blood cells, white blood cells, and platelets. In a stem cell transplant, the patients own stem cells would be killed by chemotherapy drug and then replaced by stem cells from the donor. Stem cells can be collected from the bone marrow, peripheral blood or umbilical cords. In this study, umbilical cords will be the source of the stem cells.
Currently, large inventories of umbilical cord blood units are available in public banks for transplantation in those lacking bone marrow donors. UCB transplants 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 (as compared to recipients of unrelated donor marrow and peripheral blood cells).
The two main causes of death after umbilical cord blood transplantation for disorders for these kinds of patients, are graft failure and infection.
In this study we are trying to address these two problems by using different drugs to prepare patients for the transplant.
To help improve engraftment (cells begin to grow), we will include the drug Fludarabine to the usually used Busulfan and Cytoxan that the study patients will receive before their transplant.
We will try to decrease the chance of developing graft-versus-host disease (GvHD) by using Cyclosporin A (CSA) and Mycophenolate Mofetil (MMF), instead of Anti-Thymocyte Globulin (ATG) which is normally used.
详细描述
Patients will be examined to make sure that they meet the requirements of this study. There will be tests of the heart and of the lungs. X-rays will be taken of the lungs and other organs, depending on the disease. An MRI and consultations with different specialists will also be conducted.
Patients also must have a negative pregnancy test before entering this study if they are a woman of childbearing potential. The blood will be tested for viruses and to look at the functioning of the liver and kidneys. The examination also includes HIV testing. If the patient has HIV, they will not be able to be treated on this protocol.
After we have determined that the patient is eligible for treatment on this study and a suitable UCB stem donor has been found, they will have a central line placed.
After placement of the central line, the following chemotherapy will be given to after admission to the hospital:
- 9 days before the infusion through 6 days before the infusion: Busulfan every 6 hours for 16 total doses.
- 5 days before the infusion through 2 days before the infusion: Cytoxan given daily for 4 days over 2 hours. (It can be given over 1 to 4 hours if needed as decided by the physician). Mesna will be given per standards.
- 4 days before the infusion through 1 day before the infusion: Fludarabine given daily for 4 days over 1 hour.
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 17 Years（Child）
- 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
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
干预措施: Busulfan (Drug)
Umbilical Cord Blood Transplant Treatment Plan
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
干预措施: Cytoxan (Drug)
Umbilical Cord Blood Transplant Treatment Plan
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
干预措施: Fludarabine (Drug)
Umbilical Cord Blood Transplant Treatment Plan
Busulfan, Cytoxan, Fludarabine, 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 Umbilical Cord Blood Transplant in Pediatric Patients.
时间窗: 1 year
To determine the overall survival rate at 1 year after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Overall Survival at 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
时间窗: 3 years
To determine the overall survival rate at 3 years after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients.
时间窗: 100 days
To determine the overall survival rate at 100 days after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
次要结局
- Number of Participants With Platelet Engraftment(Day 42)
- Number of Participants With Chronic GvHD(1 year)
- Number of Participants With Neutrophil Engraftment(Day 42)
- Incidence of Severe Grade III-IV Acute GvHD at Day 100.(Day 100)
- Number of Participants With Donor Engraftment After Transplant.(100 days, 6 months and 12 months)
研究者
Caridad Martinez
Assistant Professor, Pediatric Hematology/Oncology, Center for Cell and Gene Therapy
Baylor College of Medicine