NCT01128855已完成1 期
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study to Assess the Pharmacokinetics, Safety and Tolerability of Single Subcutaneous Injections of GSK2402968 in Non-ambulant Subjects With Duchenne Muscular Dystrophy
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F
研究概览
简要总结
The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 9 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a sponsor approved DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH (Comparative Genomic Hybridisation), SCAIP (Single Condition Amplification/Internal Primer) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by treatment with GSK
- •Age 9 years old or greater at Screening;
- •Non-ambulant (at least 1 year in a wheelchair) within the last 4 years;
- •Life expectancy at least three years;
- •Willingness and ability to comply with all protocol requirements and procedures;
- •QTc <450msec (based on single or average QTc value of triplicate ECGs obtained over a brief recording period). Note: QTc may be either QTcB or QTcF, machine read or manual overread;
- •Subjects must be willing to use adequate contraception (condoms or abstinence), from Screening until at least 5 months after the last dose of study drug;
- •Informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations).
排除标准
- •Any additional mutation (such as an additional missing exon for DMD) that cannot be treated with GSK2402968;
- •Current or history of liver or renal disease;
- •Acute illness within 4 weeks of anticipated administration of study medication, which may interfere with study assessments;
- •Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs, idebenone or other forms of Coenzyme Q10, within 6 months of the first administration of study medication;
- •Start of glucocorticosteroids within 6 months or non-stable use of glucocorticosteroids within 3 months of the anticipated first administration of study medication;
- •Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at Screening;
- •Symptomatic cardiomyopathy;
- •Use of alcohol from Screening through to the 1 month Follow-up visit ;
- •Any Child in Care.
研究组 & 干预措施
Cohort 1
Experimental
3 mg/kg GSK2402968 / placebo
干预措施: 3 mg/kg GSK2402968 (Drug)
Cohort 1
Experimental
3 mg/kg GSK2402968 / placebo
干预措施: Placebo (Other)
Cohort 2
Experimental
6 mg/kg GSK2402968 / placebo
干预措施: 6 mg/kg GSK2402968 (Drug)
Cohort 2
Experimental
6 mg/kg GSK2402968 / placebo
干预措施: Placebo (Other)
Cohort 3
Experimental
9 mg/kg GSK2402968 / placebo
干预措施: 9 mg/kg GSK2402968 (Drug)
Cohort 3
Experimental
9 mg/kg GSK2402968 / placebo
干预措施: Placebo (Other)
Cohort 4
Experimental
12 mg/kg GSK2402968 / placebo
干预措施: 12 mg/kg GSK2402968 (Drug)
Cohort 4
Experimental
12 mg/kg GSK2402968 / placebo
干预措施: Placebo (Other)
结局指标
主要结局
Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F
时间窗: 35 days
Incidence of Adverse Events
时间窗: 35 days
Incidence of Injection Site Reactions
时间窗: 35 days
次要结局
未报告次要终点
研究者
研究点 (1)
Loading locations...
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