A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With Maroteaux-Lamy Syndrome (MPS VI)
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 4
- 试验地点
- 4
- 主要终点
- Change in Height
研究概览
简要总结
The purpose of the study is to evaluate the safety and efficacy of two dose levels of Naglazyme in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.
详细描述
The primary objective of the study was to evaluate the efficacy of two dose levels of Naglazyme in preventing the progression of skeletal dysplasia in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.
The secondary objective of the study was to evaluate the efficacy of the two dose levels of Naglazyme in preventing several measures of disease progression in infants under the age of one year who have MPS VI by monitoring urinary GAGs, gross and fine motor function, cardiac function, vision, hearing, and use of health resources.
The safety objective of the study was to evaluate the safety of two dose levels of Naglazyme in infants under the age of one year who have MPS VI.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- — 至 1 Year（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent by a parent or legal guardian
- •Parent or legal guardian willing and able to comply with all study procedures
- •Equal to or greater than 36 weeks estimated gestational age by physical exam at birth
- •Has a diagnosis of MPS VI based on a documented prenatal diagnosis or fibroblast or leukocyte N-acetylgalactosamine 4-sulfatase (ASB) enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory
- •Is less than one year of age
- •Has no evidence of skeletal dysplasia based on physical exam
排除标准
- •Parent of legal guardian perceived to be unreliable or unavailable for study participation
- •Use of any investigational drug within 30 days prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments
- •Has concurrent disease or condition that would interfere with study participation or safety (i.e., has previously undergone hematopoietic stem cell transplantation such as bone marrow or cord blood transplantation, or major organ transplantation)
- •Any condition that, in the view of the principle investigator, renders the subject at high risk from treatment compliance and/or completing the study
- •Has known hypersensitivity to Naglazyme
- •Has previously received Naglazyme
研究组 & 干预措施
Naglazyme, 1.0 mg/kg
Dose comparison
干预措施: Naglazyme (Drug)
Naglazyme, 2.0 mg/kg
Dose Comparison
干预措施: Naglazyme (Drug)
结局指标
主要结局
Change in Height
时间窗: 52 weeks
Change in Weight
时间窗: 52 weeks
Change in Haed Circumference
时间窗: 52 weeks
次要结局
- Change in Urinary Glycosaminoglycan Levels(minimum 52 weeks of dosing)