Unrelated Donor Transplant for Malignant and Non-Malignant Disorders
2015年4月14日 更新者:Columbia University
Unrelated Donor Stem Cell Transplant for Patients With Malignant and Non-Malignant Disorders
Unrelated matched donor (cord blood, bone marrow or peripheral blood) allogeneic stem cell transplantation (UDAlloSCT) with either myeloablative or reduced intensity conditioning will be well tolerated and result in a high degree of engraftment in patients with selected malignant and non malignant disorders.
研究概览
详细说明
This is a non-randomized study to determine the tolerability and degree of engraftment of unrelated matched donor allogeneic stem cell transplantation with either myeloablative or reduced intensity conditioning in patients with selected malignant and non malignant disorders.
Patients will receive one of either full intensity or reduced intensity regimen based on the patient's disease status, organ function and performance and determined by the PI.
研究类型
介入性
注册 (实际的)
22
阶段
- 阶段2
联系人和位置
本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。
学习地点
-
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New York
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New York、New York、美国、10032
- Columbia University Medical Center
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-
参与标准
研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
不超过 55年 (孩子、成人)
接受健康志愿者
不
有资格学习的性别
全部
描述
Inclusion Criteria:
- Adequate renal function defined as: serum creatinine 2.0 x normal, or creatinine clearance or radioisotope GFR > 40 ml/min/m2 or > 40 ml/min/1.73 m2 or an equivalent GFR as determined by the institutional normal range.
- Adequate liver function defined as: total bilirubin < 2.5 x normal; or SGOT (AST) or SGPT (ALT) < 5.0 x normal.
- Adequate cardiac function defined as: shortening fraction of > 25% by echocardiogram, or ejection fraction of > 40% by radionuclide angiogram or echocardiogram.
- Adequate pulmonary function defined as: DLCO > 35% by pulmonary function test. For children who are uncooperative, no evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry > 94% in room air.
Diseases:
- CML (CP, AP or BC)
- AML/MDS/JCML
- ALL
- Lymphoma (Hodgkin's and non-Hodgkin's)
- Non-malignant disorders
Bone Marrow Failure Syndromes: Patients with the following diagnoses are eligible:
- Severe Aplastic Anemia:
- Fanconi Anemia
- Severe Congenital Neutropenia (Kostmann's Syndrome)
- Amegakaryocytic Thrombocytopenia
- Diamond-Blackfan Anemia
- Infantile Osteopetrosis
- Schwachman-Diamond Syndrome
- Dyskeratosis Congenita
- Other bone marrow failure syndromes at discretion of Principal Investigator
Immunodeficiencies:
- SCIDS, all subtypes
- Combined Immunodeficiency Syndrome
- Wiskott-Aldrich syndrome
- Chronic Granulomatous Disease
- Chediak-Higashi Syndrome
- Leukocyte Adhesion Deficiency
- Other immunodeficiencies at discretion of Principal Investigator
Inborn Errors of Metabolism (IEOM):
- Transplant is recommended for the following disorders: Hurler syndrome (L-iduronidase deficiency, MPS-I), Maroteaux-Lamy syndrome (galactosamine-4-sulfatase deficiency, MP VI), Sly syndrome (glucuronidase deficiency, MPS-VII), Globoid cell Leukodystrophy (galactocerebrosidasedeficiency), Metachromatic leukodystrophy (arylsulfatase A deficiency), Childhood-onset X-linked adrenoleukodystrophy (X-ALD), Fucosidosis (fucosidase deficiency), Mannosidosis, Aspartylglucosaminuria, Niemann-Pick Disease Type B (acid sphingomyelinase deficiency), Gaucher disease (glucocerebrosidase deficiency) Type I (non neuropathic), Other diagnoses may be considered at the discretion of the Principal Investigator
- For X-ALD patients greater than 5 years of age, IQ > 80 is required. For other patients greater than 5 years of age, IQ > 70 is required.
- For patients less than 5 years of age, the developmental quotient or clinical neurodevelopmental examination should demonstrate potential for stabilization at a level of functioning where continuous life support (e.g. mechanical ventilation) would not be predicted to be required in the year following transplantation.
Histiocytosis:
- Hemophagocytic Lymphohistiocytosis (HLH)
- Familial Erythrophagocytic Lymphohistiocytosis
- Langerhans Cell Histiocytosis
- Malignant Histiocytosis
- Other Malignant and non-malignant diseases: Other malignant and non-malignant diseases not listed above may be eligible if deemed appropriate by the Principal Investigator.
Exclusion Criteria:
- Women who are pregnant and/or breast feeding are ineligible
学习计划
本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:单组作业
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
---|---|
实验性的:UDAlloSCT + Therapy
This is a non-randomized study to test the safety and response of unrelated matched donor allogeneic stem cell transplantation (UDAlloSCT) with either myleoablative (full intensity) or reduced intensity conditioning therapy in patients with selected malignant and non-malignant disorders.
UDAlloSCT has been performed in both adults and children as an alternative transplant for patients who lack and HLA-matched family donor in both malignant and non-malignant disease with varying degrees of response.
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unrelated matched donor allogeneic stem cell transplantation (UDAlloSCT)
Full Intensity Therapy (myeloablative) (TBI + Thiotepa + Cyc) OR Reduced Intensity Therapy (Fludarabine, Busulfan, and Alemutuzumab (FBA))
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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
---|---|---|
Incidence of toxicity related to myeloablative therapy
大体时间:Up to 10 years from start of study
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To determine the safety and toxicity of myeloablative therapy (TBI + Melphalan) and unrelated donor alloSCT in selected patients with malignant and non-malignant disorders.
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Up to 10 years from start of study
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
---|---|---|
Incidence of toxicity related to reduced intensity therapy
大体时间:Up to 10 years from start of study
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To determine the safety and toxicity of reduced intensity therapy (Fludarabine, Busulfan, and Alemtuzumab (FBA) and unrelated donor alloSCT in selected patients with malignant and non malignant disorders
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Up to 10 years from start of study
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Percentage of donor chimerism
大体时间:Up to 10 years from start of study
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To quantitate the percentage of donor chimerism following both myeloablative and reduced intensity conditioning and unrelated donor alloSCT in selected patients with malignant and non-malignant disorders.
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Up to 10 years from start of study
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Prevalence of progression free survival
大体时间:Up to 10 years from start of study
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To estimate the progression free survival (PFS), if applicable, event free survival (EFS) and overall survival (OS) following unrelated donor alloSCT in selected patients with malignant and non malignant disorders.
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Up to 10 years from start of study
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
调查人员
- 首席研究员:Mitchell S Cairo, MD、Columbia University
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始
2002年11月1日
初级完成 (实际的)
2011年4月1日
研究完成 (实际的)
2011年4月1日
研究注册日期
首次提交
2008年6月3日
首先提交符合 QC 标准的
2010年1月14日
首次发布 (估计)
2010年1月15日
研究记录更新
最后更新发布 (估计)
2015年4月15日
上次提交的符合 QC 标准的更新
2015年4月14日
最后验证
2015年4月1日
更多信息
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