Anti-CD45 (YTH-24 & YTH 54) and ANTI-CD52 (CAMPATH-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Stem Cell Transplantation of Patients With Inherited Metabolic Storage Diseases
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 2
研究概览
简要总结
This study treats patients with an inherited disease that prevents the body from making a specific protein or enzyme needed for the body's metabolism. Lack of this enzyme causes accumulation of harmful or toxic substances in the body, which leads to deterioration and failure of organs such as the brain or the heart. This disease can be fatal.
Some patients with inherited metabolic storage disease may benefit from an allogeneic stem cell transplant ('allogeneic' means that the stem cells come from another person). Stem cells are created in the bone marrow. They mature into different types of blood cells that are needed including red blood cells, white blood cells, and platelets. Stem cells, when transplanted, can make a new blood system. Donor stem cells can make the protein or enzyme patients with this disease cells cannot. The donor cells may prevent further accumulation of toxic substances. It is hoped that the donor cells can prevent or stop the disease from progressing.
This research study uses a new pre-treatment combination of two drugs, Anti-CD45 and CAMPATH-1H. Anti-CD45 and CAMPATH-1H are antibodies against certain types of blood cells. CAMPATH-1H is particularly important because it stays active in the body for a long time after infusion, which means it may work longer at preventing GVHD symptoms. In addition to antibodies, patients will receive Fludarabine, which is a chemotherapy drug. Fludarabine kills bone marrow cells and is given to reduce the bone marrow cells so that donor stem cells may 'take.'
详细描述
Fludarabine will be given as a daily IV (intravenous, by vein) infusion for a total of 5 days. CAMPATH-1H will be given as a daily 4-hour IV (intravenous, by vein) infusion for three days. Anti-CD45 will be given as a daily 6-hour IV infusion over the next 4 days. Then patients will have a one-day rest period before receiving the stem cell transplant. To help prevent the body from rejecting the transplant, patients will also receive the drug FK506, starting two days before the transplant and continuing for three months. If there is no GVHD, the amount of FK506 patients are taking will be reduced by 20% every 2 weeks until this medication is stopped.
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
Fludarabine, CAMPATH-1H , Anti-CD45, FK506
Fludarabine will be given as a daily IV (intravenous, by vein) infusion for a total of 5 days. CAMPATH-1H will be given as a daily 4-hour IV (intravenous, by vein) infusion for three days. Anti-CD45 will be given as a daily 6-hour IV infusion over the next 4 days. To help prevent body from rejecting the transplant, the drug FK506 will be given, starting two days before the transplant and continuing for three months.
干预措施: CAMPATH-1H (Drug)
Fludarabine, CAMPATH-1H , Anti-CD45, FK506
Fludarabine will be given as a daily IV (intravenous, by vein) infusion for a total of 5 days. CAMPATH-1H will be given as a daily 4-hour IV (intravenous, by vein) infusion for three days. Anti-CD45 will be given as a daily 6-hour IV infusion over the next 4 days. To help prevent body from rejecting the transplant, the drug FK506 will be given, starting two days before the transplant and continuing for three months.
干预措施: Anti-CD45 (Drug)
Fludarabine, CAMPATH-1H , Anti-CD45, FK506
Fludarabine will be given as a daily IV (intravenous, by vein) infusion for a total of 5 days. CAMPATH-1H will be given as a daily 4-hour IV (intravenous, by vein) infusion for three days. Anti-CD45 will be given as a daily 6-hour IV infusion over the next 4 days. To help prevent body from rejecting the transplant, the drug FK506 will be given, starting two days before the transplant and continuing for three months.
干预措施: FK506 (Drug)
Fludarabine, CAMPATH-1H , Anti-CD45, FK506
Fludarabine will be given as a daily IV (intravenous, by vein) infusion for a total of 5 days. CAMPATH-1H will be given as a daily 4-hour IV (intravenous, by vein) infusion for three days. Anti-CD45 will be given as a daily 6-hour IV infusion over the next 4 days. To help prevent body from rejecting the transplant, the drug FK506 will be given, starting two days before the transplant and continuing for three months.
干预措施: Fludarabine (Drug)
研究者
Robert Krance
Professor
Baylor College of Medicine