NCT01372228终止1 期
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
适应症
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 2
- 主要终点
- Production of missing enzyme at levels greater than or equal to 10% of normal
研究概览
简要总结
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
详细描述
The objective for the study is to establish chimerism following reduced intensity conditioning with no grade III/IV GVHD. The primary endpoint we will follow is production of the missing enzyme at ≥ 10% of the normal level at day 180 post-transplant in > 90% of patients.
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
- 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
结局指标
主要结局
Production of missing enzyme at levels greater than or equal to 10% of normal
时间窗: Day 180 post transplant to three years
次要结局
- Enriched Hematopoetic Stem Cell Engraftment(One month to three years)
研究者
研究点 (2)
Loading locations...
相似试验
已完成
1 期
Phase I/II Pilot Study of Mixed Chimerism to Treat HemoglobinopathiesSickle Cell DiseaseNCT00777231Talaris Therapeutics Inc.9
撤回
1 期
Phase I/II Pilot Study of Mixed Chimerism to Treat HemoglobinopathiesAnemia, Sickle CellComplex and Transfusion-dependent HemoglobinopathiesDiamond-Blackfan AnemiaBone Marrow Failure SyndromesAlpha-ThalassemiaBeta-ThalassemiaThalassemiaNCT01419704Talaris Therapeutics Inc.
撤回
1 期
Allogeneic Stem Cell Transplantation for the Treatment of Multiple SclerosisMultiple SclerosisNCT00497952Talaris Therapeutics Inc.
已完成
1 期
Induction of Donor Specific Tolerance in Recipients of Cardiac Allografts by Donor Stem Cell InfusionHeart TransplantationNCT00497757Suzanne T. Ildstad62
进行中（未招募）
1 期
Induction of Donor Specific Tolerance in Recipients of Live Donor Kidney Allografts by Donor Stem Cell InfusionRenal FailureNCT00498160Talaris Therapeutics Inc.38