NCT03367546终止2 期
Haploidentical Donor T-cell Replete Allogeneic Hematopoietic Cell Transplant Following Reducing Intensity Conditioning for Patients With Selected High Risk Non-Malignant Disease
适应症
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 5
- 试验地点
- 2
- 主要终点
- Neutrophil Recovery
研究概览
简要总结
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who lack a suitable HLA-matched sibling donor.
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
- 0 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Sickle Cell Disease (SCD)
- •* If diagnosis of SCD must meet one or more of the following disease characteristics:
- •Stroke, CNS hemorrhage or a neurologic event lasting longer than 24 hours, or abnormal cerebral MRI or cerebral arteriogram or MRI angiographic study and impaired neuropsychological testing
- •Acute chest syndrome with a history of recurrent hospitalizations or exchange transfusions
- •Recurrent vaso-occlusive pain 3 or more episodes per year for 3 years or more years or recurrent priapism,
- •Impaired neuropsychological function and abnormal cerebral MRI scan
- •Stage I or II sickle lung disease,
- •Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate [GFR] 30-50% of the predicted normal value)
- •Bilateral proliferative retinopathy and major visual impairment in at least one eye
- •Osteonecrosis of multiple joints with documented destructive changes
- •Requirement for chronic transfusions
- •RBC alloimmunization
- •Transfusion Dependent Alpha- or Beta-Thalassemia
- •Other Non-Malignant Hematologic Disorders:
- •Transfusion dependent or involve other potential life-threatening cytopenias, including but not limited to Paroxysmal Nocturnal Hemoglobinuria, Glanzmann's Thrombasthenia, Severe Congenital Neutropenia and Shwachman-Diamond Syndrome
- •Diagnosis of ALD by abnormal plasma very long chain fatty acid (VLCFA) profile or ABCD1 gene mutation
- •Cerebral disease on MRI
- •Absence of a Major Functional Disability (cortical blindness, loss of communication, wheelchair dependence) on the ALD Neurologic Function Scale
- •Other inherited metabolic disorders:
- •Any other inherited metabolic disorder for which alloHCT is indicated and for whom, in the opinion of the treating physician, the patient's best treatment option is with a haploidentical donor following non-myeloablatve conditioning.
- •Age, Performance Status, Consent
- •Age: 0-55 years
- •Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70
- •Consent: voluntary written consent (adult or parental/guardian)
- •Adequate Organ Function
- •Renal: Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
- •Hepatic: Bilirubin and ALT <3 times the upper limit of institutional normal
- •Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%.
排除标准
- •Availability of a suitable HLA-matched related donor
- •Uncontrolled infection
- •Pregnant or breastfeeding
- •HIV positive
结局指标
主要结局
Neutrophil Recovery
时间窗: Day 42
Incidence of neutrophil recovery by day +42
次要结局
- Overall Survival (OS)(1 year)
- Primary Graft Failure (neutropenic and non-neutropenic)(Day 42)
研究者
研究点 (2)
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