Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc)
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Toxicity
研究概览
简要总结
The purpose of this study is to determine if a reduced intensity (RI) (non-myeloablative) chemoimmunotherapy followed by Allogeneic Stem Cell Transplantation AlloSCT (matched family donors and matched unrelated cord blood donors) will be well tolerated.
详细描述
This is to test whether a reduced intensity will result in a high degree of mixed or complete donor chimerism and stabilization of autoimmune disease in a select group of patients with medically refractory SLE or SSc.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 7 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diffuse Systemic Sclerosis and variants as per ACR criteria
- •Medically refractory disease
- •Adequate Organ Function - Pulmonary function
- •Renal function, Cardiac function defined as:
- •SGOT (AST) or SGPT (ALT) <5 x upper limit of normal
- •Diagnosis of SLE - Medically refractory disease
排除标准
- •Karnofsky/Lansky <60%
研究组 & 干预措施
Reduced Intensity Regimen for Refractory SLE
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).
干预措施: Reduced Intensity Allogeneic Transplant (Procedure)
Reduced Intensity Regimen for Refractory SLE
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).
干预措施: Fludarabine (Drug)
Reduced Intensity Regimen for Refractory SLE
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).
干预措施: Busulfan (Drug)
Reduced Intensity Regimen for Refractory SLE
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).
干预措施: Campath (Drug)
Reduced Intensity Regimen for SSc
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).
干预措施: Reduced Intensity Allogeneic Transplant (Procedure)
Reduced Intensity Regimen for SSc
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).
干预措施: Fludarabine (Drug)
Reduced Intensity Regimen for SSc
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).
干预措施: Busulfan (Drug)
Reduced Intensity Regimen for SSc
RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).
干预措施: Campath (Drug)
结局指标
主要结局
Toxicity
时间窗: 1 year
Toxicity associated with reduced intensity regimen of fludarabine/busulfan and Campath followed by allogeneic stem cell transplant in patients with medically refractory Systemic Lupus Erythematosus (SLE) or SSc is measured.
次要结局
- Chimerism(1 year)
- Immune Reconstitution.(1 year)
- Progression Free and Overall Survival.(1 year)
研究者
Mitchell Cairo
Principal Investigator
New York Medical College