Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- Number of Patients With Detectable Collagen Type VII
研究概览
简要总结
RATIONALE: In animal models, stem cells have been shown to home to the skin and repair the biochemical and structural abnormalities associated with recessive dystrophic epidermolysis bullosa (RDEB) (collagen 7 deficiency).
PURPOSE: To determine the safety and effectiveness of stem cell infusion in the treatment of RDEB.
详细描述
OBJECTIVES:
Primary
- Estimate the incidence of detectable donor-derived collagen type VII at day 100 in patients with epidermolysis bullosa by donor.
Secondary
- Determine the incidence of transplant-related mortality at day 180
- Determine the incidence of blood chimerism at days 21, 100, 180, 365, and 730
- Determine the incidence of neutrophil recovery at day 42 and platelet recovery at day 180
- Determine the incidence of acute graft-versus-host disease (GVHD) grade II-IV and grade III-IV at day 100
- Determine the incidence of chronic GVHD at 1 year
- Determine the probability of survival at 1 and 2 years
- Determine the incidence of donor derived cells in the skin
- Determine resistance to blister formation OUTLINE: This is an open-label, pilot study.
- Conditioning regimen: Busulfan intravenously (IV) over 2 hours every 6 hours on days -9 to -4, fludarabine phosphate IV over 1 hour on days -5 to -3, and high-dose cyclophosphamide IV over 1 hour on days -5 to -2.
- Stem cell transplantation on day 0.
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 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Epidermolysis Bullosa (EB) Patients
Epidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant.
干预措施: busulfan (Drug)
Epidermolysis Bullosa (EB) Patients
Epidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant.
干预措施: cyclophosphamide (Drug)
Epidermolysis Bullosa (EB) Patients
Epidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant.
干预措施: fludarabine phosphate (Drug)
Epidermolysis Bullosa (EB) Patients
Epidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant.
干预措施: hematopoietic bone marrow transplantation (Procedure)
结局指标
主要结局
Number of Patients With Detectable Collagen Type VII
时间窗: Day 100 Post Transplant
Number of patients with epidermolysis bullosa who had collagen type VII. Type VII collagen defects cause recessive dystrophic epidermolysis bullosa (RDEB), a blistering skin disorder often accompanied by epidermal cancers.
次要结局
- Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD)(Day 365 Post Transplant)
- Number of Patients With Resistance to Blister Formation(Month 1 through Month 24 Inclusive)
- Number of Patients With >70% Donor Chimerism(Days 21, 100, 180, 365 and 730 Post Transplant)
- Number of Patients With Platelet Engraftment(Day 180 Post Transplant)
- Number of Patients With Transplant-Related Mortality(Day 180 Post Transplant)
- Overall Survival(1 year and 2 years Post Transplant)
- Number of Patients With Acute Graft-Versus-Host Disease (GVHD)(Day 100 Post Transplant)
- Number of Patients With Donor Derived Cells in Skin(Day 90 Post Transplant)
- Number of Patients With Neutrophil Engraftment(Day 42 Post Transplant)