EUCTR2005-003512-30-PT进行中（未招募）不适用
A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) - N/A
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 4
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •In the case of subjects under the age of 18, provide written assent (if required) and written informed consent by a parent of legal guardian after the nature of the study has been explained and prior to any research-related procedures.
- •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.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Perceived to be unreliable or unavailable for study participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unavailable.
- •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.
- •Concurrent disease or condition that would interfere with study participation or safety (i.e., has previously undergone haematopoietic stem cell transplantation such as bone marrow transplantation, or major organ transplantation).
- •Any condition that, in the view of the PI, renders the subject at high risk from treatment compliance and/or completing the study.
- •Has known hypersensitivity to Naglazyme.
- •Has previously received Naglazyme.
研究者
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