A Multi-center, Open-Label Extension Study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) Treatment in Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Shire
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- Days of Exposure to HGT-1111
研究概览
简要总结
This is a multi-center, open-label, extension study of patients with late infantile MLD who have previously completed clinical study HGT-MLD-048 (NCT00633139), defined as the completion of all Week 52 procedures. This group of patients will be offered ongoing treatment with HGT-1111 in this protocol. One infusion will be given every other week until the product is commercially available, the patient discontinues, or the study is terminated by the Sponsor, provided no safety issues have emerged.
详细描述
The primary objective of this study is to provide ongoing treatment of HGT-1111 to patients who have completed study HGT-MLD-048 (previously study rhASA-03 - NCT00633139) until HGT-1111 is commercially available or the study is terminated by the Sponsor, provided no safety concerns have emerged. The secondary objective of this study is to monitor disease progression and the safety profile of HGT-1111 administered to patients who have completed study HGT-MLD-048 (NCT00633139).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (Trial-related activities are any procedures that would not have been performed during normal management of the subject)
- •Completion of study HGT-MLD-048 (NCT00633139)
- •The subject and his/her guardian(s) must have the ability to comply with the protocol
排除标准
- •Spasticity so severe to inhibit transportation
- •Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition that, in the opinion of the Investigator, would preclude participation in the trial
- •Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
研究组 & 干预措施
HGT-1111 100 U/kg
干预措施: HGT-1111 (Drug)
HGT-1111 200 U/kg
干预措施: HGT-1111 (Drug)
结局指标
主要结局
Days of Exposure to HGT-1111
时间窗: Baseline until end of study (Week 139)
End of study was defined as until HGT-1111 was commercially available, the participant's participation was discontinued, or the study was terminated by the Sponsor.
次要结局
- Level of White Matter Metabolites(Baseline until end of study (Week 139))
- Level of Cerebrospinal Fluid (CSF) Sulfatide(Baseline until end of study (Week 139))
- Score of Gross Motor Function Measurement (GMFM)(Baseline until end of study (Week 139))