Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 13
- 试验地点
- 1
- 主要终点
- Identify covariates that impact drug exposure
研究概览
简要总结
This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH undergoing laronidase therapy and a stem cell transplant.
Sixteen patients will be enrolled over a 24 month period.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 0 Years 至 3 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Between 0 to 3 years of age
- •Meet protocol specific eligibility criteria for allogeneic HCT for MPS IH
- •Planning to receive laronidase both pre and post-transplant in an inpatient setting as part of standard-of-care treatment. Virtually all patients with MPSIH being considered for transplantation at the University of Minnesota are already receiving enzyme infusions, and it is standard practice to continue to give enzyme infusions to 8 weeks post-transplant. Therefore, participation will not modify the treatment course.
排除标准
- •Patient's parent/ legal guardians are unable to provide informed consent.
研究组 & 干预措施
Collect blood from patients with MPS-IH) undergoing laronidase therapy and stem cell transplant.
The primary aim is to characterize the PK of IV laronidase in individuals with MPS IH and identify patient specific covariates that impact drug exposure. The secondary aim is to identify key differences pre-and post-HCT leading to variability in PK parameters for patients receiving IV laronidase therapy for the treatment of MPS-IH.
干预措施: Laronidase therapy and a stem cell transplant (Drug)
结局指标
主要结局
Identify covariates that impact drug exposure
时间窗: 2 years
Measure indicators for body size and maturation contribute to variability in laronidase exposure in patients with MPS IH.
Identify key differences pre- and post-HCT leading to variability in PK parameters
时间窗: 2 years
Measure endogenous source of enzyme present in relation to the transplanted cells.
次要结局
未报告次要终点