Unrelated Donor Stem Cell Transplant for Patients With Malignant and Non-Malignant Disorders
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 22
- 试验地点
- 1
- 主要终点
- Incidence of toxicity related to myeloablative therapy
研究概览
简要总结
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.
详细描述
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 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
- •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.
排除标准
- •Women who are pregnant and/or breast feeding are ineligible
研究组 & 干预措施
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.
干预措施: UDAlloSCT (Procedure)
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.
干预措施: Therapy (Other)
结局指标
主要结局
Incidence of toxicity related to myeloablative therapy
时间窗: Up to 10 years from start of study
To determine the safety and toxicity of myeloablative therapy (TBI + Melphalan) and unrelated donor alloSCT in selected patients with malignant and non-malignant disorders.
次要结局
- Percentage of donor chimerism(Up to 10 years from start of study)
- Prevalence of progression free survival(Up to 10 years from start of study)
- Incidence of toxicity related to reduced intensity therapy(Up to 10 years from start of study)