Pilot Study of Administration of Intravenous Laronidase Following Allogeneic Transplantation for Hurler Syndrome
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- Percentage of Adherence to the Scheduled Weekly Infusion by the Participants
研究概览
简要总结
This is a single center pilot study in which Laronidase will be given weekly for two years in patients with Hurler syndrome, also known as mucopolysaccharide IH (MPS I, Hurler syndrome), that have previously been treated with an allogeneic transplant.
详细描述
This 2-year open-label pilot study of laronidase includes patients (age 5-13 years) who are at least 2 years post-hematopoietic cell transplantation (HCT) and donor engrafted. Outcomes are assessed semi-annually and compared to historic controls. Eligible patients will receive Laronidase as an infusion over several hours once a week at a local site. The dosing of enzyme will be the standard doses recommended by Genzyme.
The findings of this Pilot Study will be used to assess whether a subsequent larger study can be conducted.
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 14 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant >2 years previously
- •Age <14 years old
- •>10% engrafted based on recent testing (<4 months prior to enrollment)
- •Willing to commit to traveling to the University of Minnesota every 6 months
- •Written informed consent prior to the performance of any study related procedures
排除标准
- •Previous administration of Laronidase enzyme > 3 months post transplantation
- •Anticipated survival less than 2 years
- •History of cardiac or pulmonary insufficiency, including an ejection fraction (EF) < 40% or those requiring continuous supplemental oxygen
研究组 & 干预措施
Laronidase After Transplantation
Patients with Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant >2 years previously and treated with Laronidase weekly for 2 years after transplant.
干预措施: Laronidase (Drug)
结局指标
主要结局
Percentage of Adherence to the Scheduled Weekly Infusion by the Participants
时间窗: 24 months
To determine the feasibility of giving weekly Laronidase for 2 years in patients with Hurler syndrome after allogeneic transplantation, compliance throughout the study with drug administration, the percentage of adherence to the scheduled weekly infusion for each participant is measured.
Number of Participants Experiencing Severe Adverse Events
时间窗: 24 months
Number of participants experiencing severe adverse events that occur after administration with Laronidase to determine the feasibility of giving weekly Laronidase
次要结局
- Changes in Growth Velocity(Baseline, Month 24)
- Change in Muscle Strength(Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months)
- Change in Peak Heart Rate to Monitor "Fitness"(Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months)
- Number of Participants Showing Improvements in Joint Range of Motion (ROM)(Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months)
- Shortening Fraction to Determine Systolic Cardiac Function(Baseline and month 24)
- Number of Participants With Changes in Cardiac Echo Structural Parameters(Baseline and month 24)
- Correlation of 6 Minute Walk Test With Anti-laronidase Antibody + Status(Assessed from baseline every 6 months through 2 years; change between baseline and 24 months reported.If baseline and/or 24-month data were not available, the longest interval between measurements was reported, with a minimum requirement of 12 months)