EUCTR2005-003512-30-FR进行中（未招募）1 期
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
- 1 期
- 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 or 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 hematopoietic stem cell transplantation such as bone marrow or cord blood 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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