A Phase II Trial of Busulfan, Melphalan, and Fludarabine With Peri-transplant Palifermin, Followed by a T-Cell Depleted Hematopoietic Stem Cell Transplant From HLA Matched or Mismatched Related or Unrelated Donors in Patients With Advanced Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 64
- 试验地点
- 1
- 主要终点
- To Reduce the Early Transplant-related Mortality.
研究概览
简要总结
This study will see if the researchers can lower that risk by giving the patient Palifermin. This drug helps protect the lining of the mouth, throat, and stomach. These areas typically get sores or ulcers while the blood cell counts are very low. The patient can get infections in or from these sores. Palifermin might also help the immune system recover faster. It is currently approved for patients who receive their own stem cells. That is called an autologous transplant. This study will test the use of Palifermin for T-cell depleted allogeneic stem cell transplants.
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 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients should be < 65 years. Patients > or equal to 65 years will be accrued on a case by case basis after discussion and approval by the BMT Service.
- •Patients may be of either gender or any ethnic background.
- •Patients must have a Karnofsky or Lansky Performance Status > or equal to 70%.
- •Patients must have adequate organ function measured by:
- •* Cardiac: asymptomatic or if symptomatic then LVEF at rest must be > or equal to 50% and must improve with exercise.
- •Hepatic: < 3x ULN ALT and < 1.5 total serum bilirubin, unless there is congenital benign hyperbilirubinemia.
- •Renal: serum creatinine < than or equal to 1.2 mg/dl or if serum creatinine is outside the normal range, then CrCl > 60-ml/min/1.73 m2
- •Pulmonary: asymptomatic or if symptomatic, DLCO > 50% of predicted (corrected for hemoglobin)
- •Each patient must be willing to participate as a research subject and must sign an informed consent form.
- •Parent or legal guardians of patients who are minors will sign the informed consent form.
排除标准
- •Active CNS or skin leukemic involvement
- •Female patients who are pregnant or breast-feeding
- •Active viral, bacterial or fungal infection
- •Patient seropositive for HIV-I/II; HTLV -I/II
- •Patients who have undergone a prior allogeneic or autologous stem cell transplant within the previous six months.
- •Patients who have had a previous malignancy that is not in remission.
研究组 & 干预措施
1
This is a single arm phase II trial to assess the efficacy (decrease the transplant related mortality) and safety of peri-transplant Palifermin in combination with a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with advanced MDS and AML evolved from MDS. The addition of Palifermin is to decrease the toxicity and the infection rate associated with this regimen and transplant type and to foster earlier immune reconstitution.
干预措施: Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant (Drug)
结局指标
主要结局
To Reduce the Early Transplant-related Mortality.
时间窗: conclusion of study
次要结局
- To Improve the Quality of Immune Reconstitution Following Transplantation.(conclusion of study)
- To Reduce the Incidence Rate of Fatal Post Transplant Infectious Complications.(conclusion of study)