Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Number of Patients Requiring Ventilator Support at One Year Post Transplant
研究概览
简要总结
The investigators hypothesize that weekly infusions of Laronidase ERT for 10-12 weeks prior to transplant and 8 weeks following transplant will result in a reduction of glycosaminoglycans (GAG) burden that is associated with decreased complications following transplant.
详细描述
Subjects will receive laronidase once a week intravenously for 10-12 weeks prior to transplant and for approximately 8 weeks after transplant. Laronidase will be given by intravenous infusion (IV) through a catheter and from there to your child's body's cells and organs to break down the glycosaminoglycans (GAG) buildup.
Prior to starting ERT, subjects will have a complete physical examination, which includes a complete assessment of your child's airway and lungs. In addition to standard treatment evaluations and tests, which are done prior to hematopoietic stem cell transplant (HSCT), subjects will have the following tests: an additional teaspoon of blood for a baseline test for serum antibodies against laronidase, before and after the fourth dose of laronidase, the investigators will collect 2 teaspoons of blood for an alpha-L-iduronidase enzyme level; to watch for side effects to laronidase and the development of antibodies to laronidase, approximately 2 teaspoons of blood will be collected every 3 weeks while the subject is receiving laronidase ERT.
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 7 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are candidates for first hematopoietic stem cell transplant (HSCT) according to a University of Minnesota myeloablative HSCT protocol.
排除标准
- •Not being considered for University of Minnesota myeloablative HSCT protocol.
- •Previous administration of laronidase enzyme
- •Second or subsequent HSCT.
研究组 & 干预措施
Laronidase ERT Treatment
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
干预措施: Stem Cell Transplant (Procedure)
Laronidase ERT Treatment
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
干预措施: Laronidase ERT (Drug)
结局指标
主要结局
Number of Patients Requiring Ventilator Support at One Year Post Transplant
时间窗: one year
Number of Patients Alive at One Year Post Transplant
时间窗: one year
次要结局
- Donor Engraftment(Day 100 post transplant)
- Patients With Grade III-IV Acute GVHD(Day 100 post transplant)
- Reduction in Glycosaminoglycans (GAG)(Prior to, During and After ERT)
- Toxicity (Adverse Events) Associated With Infusions of Laronidase(1 year post transplant)
- Development of Anti-iduronidase Antibodies in Serum(1 Year)
- Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography(Baseline, 12 weeks after laronidase, after transplant)