NCT01572636终止不适用
MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Overall Survival
研究概览
简要总结
This is a standard of care treatment guideline for patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are being considered as candidates for first hematopoietic stem cell transplantation (HSCT) according to a University of Minnesota myeloablative HSCT protocol.
详细描述
Laronidase Enzyme Replacement Therapy will be performed using laronidase once a week for 12 weeks prior to hematopoietic stem cell transplantation and for 8 weeks post-transplant to reduce pulmonary complications.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) and being considered as a candidate for first transplant according to a University of Minnesota myeloablative hematopoietic stem cell transplant (HSCT) protocol
排除标准
- •No prior therapy with laronidase enzyme replacement therapy (ERT)
研究组 & 干预措施
Laronidase use in Hurler Syndrome
Laronidase receiving prior and post transplant
干预措施: Laronidase (Drug)
结局指标
主要结局
Overall Survival
时间窗: At 1 Year
Patients alive at 1 year post transplantation.
次要结局
- Incidence of Engraftment(1 Year Post Transplant)
- Incidence of Grade III-IV Acute Graft Versus Host Disease(Day 100)
- Proportion of patients in need of ventilator support(1 Year)
研究者
研究点 (1)
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