NCT01480245终止3 期
An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 233
- 试验地点
- 1
- 主要终点
- Differences between the 6MWD at baseline and Week 104
研究概览
简要总结
The purpose of this study is to explore long-term safety, tolerability and efficacy of GSK2402968 in DMD subjects who previously participated in either DMD114117 or DMD114044.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Previous participation in either DMD114117 or DMD114044
- •Continued use of glucocorticoids
- •Willing and able to comply with all protocol requirements
- •Able to give informed consent
- •French subjects: Eligible for inclusion only if either affiliated to or a beneficiary of a social security category.
排除标准
- •Subject experienced a serious adverse event or who met safety stopping criteria that remains unresolved from DMD114117 or DMD114044, which in opinion of the investigator could have been attributable to study medication and is ongoing,
- •Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs,except GSK2402968, within 1 month of the first administration of study medication,
- •Current or anticipated participation in any investigational clinical studies,
- •History of significant medical disorder which may confound the interpretation of either efficacy or safety data e.g. current history of renal or liver disease/impairment, history of inflammatory disease.
研究组 & 干预措施
Continuous Dosing
Experimental
GSK2402968 6mg/kg/week
干预措施: GSK2402968 (Drug)
Intermittent Dosing
Experimental
GSK2402968 6mg/kg/week
干预措施: GSK2402968 (Drug)
结局指标
主要结局
Differences between the 6MWD at baseline and Week 104
时间窗: 104 weeks
次要结局
- Pediatric Quality of Life Neuromuscular module(104weeks)
- Time to major disease milestones(104 weeks)
- Timed Function tests(104 weeks)
- Muscle strength(104 weeks)
- North Star Ambulatory Assessment Scores(104 weeks)
- Functional Outcomes Assessment(104 weeks)
- Creatine kinase Serum concentrations(104 weeks)
- Pulmonary Function(104 weeks)
- Frequency of accidental falls during 6 Minute Walk Distance test(104 weeks)
- Clinician Global Impression of Improvement(104 weeks)
- Health Utilities Index(104 weeks)
研究者
研究点 (1)
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