临床试验/NCT00630253

NCT00630253已完成1 期

A Study of Cyclophosphamide, Fludarabine, and Antithymocyte Globulin Followed by Matched Sibling Donor Hematopoietic Cell Transplantation in Patients With Fanconi Anemia

Masonic Cancer Center, University of Minnesota1 个研究点分布在 1 个国家目标入组 31 人开始时间: 2000年2月17日最近更新: 2021年10月12日

适应症

干预措施

试验速览

阶段: 1 期
状态: 已完成
发起方: Masonic Cancer Center, University of Minnesota
入组人数: 31
试验地点: 1
主要终点: Number of Participants Experiencing Graft Failure

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

RATIONALE: Giving chemotherapy, such as cyclophosphamide and fludarabine, before a donor stem cell transplant helps to remove the patient's cells to allow for the transplant cells to take and grow. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells can make an immune response against the body's normal cells. Giving antithymocyte globulin and removing the T cells from the donor cells before transplant and giving cyclosporine before and after transplant may stop this from happening.

PURPOSE: This phase I/II trial is studying the side effects of cyclophosphamide, fludarabine, and antithymocyte globulin followed by donor stem cell transplant and to see how well it works in treating patients with Fanconi anemia.

详细描述

OBJECTIVES:

Primary

To determine the probability of engraftment in patients with Fanconi anemia treated with cyclophosphamide, fludarabine phosphate, and antithymocyte globulin followed by HLA-genotypically identical sibling donor hematopoietic stem cell transplantation that is T-cell depleted.

Secondary

To evaluate the incidence of acute graft-versus-host disease (GVHD) and chronic GVHD in patients treated with this regimen.
To evaluate the incidence of regimen-related toxicity in these patients.
To evaluate the 1-year survival of patients treated with this regimen.
To evaluate the incidence of late secondary malignancies (e.g., squamous cell carcinoma of the head and neck or cervix) in patients treated with this regimen.

研究设计

研究类型: Interventional
分配方式: Non Randomized
干预模型: Single Group
主要目的: Treatment
盲法: None

入排标准

年龄范围: — 至 59 Years（Child, Adult）
性别: All
接受健康志愿者: 否

入选标准

•Patients must be <60 years of age with a diagnosis of Fanconi Anemia (FA).
•Patients must have an HLA-A, B, DRB1 identical sibling donor. Patients and donors will be typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing.
•Patients with FA must have moderately severe aplastic anemia (AA), early myelodysplastic syndrome (MDS) with no excess blasts with or without chromosomal abnormalities.
•In patients <18 years of age, moderately severe aplastic anemia is defined as having at least one of the following:
•platelet count <40 x 10^9/L
•absolute neutrophil count (ANC) <10 x 10^8/L
•Hgb <9 g/dL
•In patients 18-60 years of age, moderately severe aplastic anemia is defined as having at least one of the following:
•platelet count <20 x 10^9/L
•absolute neutrophil count ANC <5 x 10^8/L
•Hgb <8 g/dL
•Early myelodysplastic syndrome, with multilineage dysplasia with < 5% blasts, with or without chromosomal anomalies.
•Adequate major organ function including:
•Cardiac: ejection fraction >45%
•Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
•Karnofsky performance status >70% or Lansky >50%
•Women of child bearing age must be using adequate birth control and have a negative pregnancy test.

排除标准

•Active bacterial infection within one week of hematopoietic cell transplant (HCT)
•Active fungal infection at time of HCT.
•Late MDS with greater than 5% blasts in bone marrow.
•Acute myelogenous leukemia (AML) or history of AML
•Malignant solid tumor (e.g. squamous cell carcinoma of the head/neck/cervix) within 2 years of HCT.
•Pregnant or lactating female.

研究组 & 干预措施

Marrow Isolex

Experimental

bone marrow processed using Isolex 300i (for patients enrolled through April 2010)

干预措施: Anti-Thymocyte Globulin (Biological)

Marrow Isolex

Experimental

bone marrow processed using Isolex 300i (for patients enrolled through April 2010)

干预措施: Cyclophosphamide (Drug)

Marrow Isolex

Experimental

bone marrow processed using Isolex 300i (for patients enrolled through April 2010)

干预措施: Fludarabine (Drug)

Marrow Isolex

Experimental

bone marrow processed using Isolex 300i (for patients enrolled through April 2010)

干预措施: Hematopoietic Stem Cell Transplantation (Procedure)

Marrow Isolex

Experimental

bone marrow processed using Isolex 300i (for patients enrolled through April 2010)

干预措施: Methylprednisolone (Drug)

Marrow Isolex

Experimental

bone marrow processed using Isolex 300i (for patients enrolled through April 2010)

干预措施: Filgrastim (Drug)

Marrow Isolex

Experimental

bone marrow processed using Isolex 300i (for patients enrolled through April 2010)

干预措施: Cyclosporine (Drug)

Marrow Isolex

Experimental

bone marrow processed using Isolex 300i (for patients enrolled through April 2010)

干预措施: Mycophenolate Mofetil (Drug)