EUCTR2010-018412-32-GB进行中（未招募）不适用
A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy
GlaxoSmithKline Research and Development LTD0 个研究点目标入组 54 人开始时间: 2010年4月16日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 54
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
- Male
入选标准
- •1.Ambulant subjects with Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a state-of-the-art 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 GSK2402968-induced DMD exon 51 skipping,
- •2.Aged at least 5 years,
- •4.Life expectancy of at least 1 year,
- •5.Able to rise from floor in =7 seconds (without aids/orthoses),
- •6.Able to complete the 6MWD test with a distance of at least 75m. In addition, results of 6MWD must be within 20% of each other at each pre-drug visit,
- •7.Receiving glucocorticoids for a minimum of 6 months immediately prior to screening, with no significant change in total daily dosage or dosing regimen for a minimum of 3 months immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly for the duration of the study,
- •8.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, and machine read or manual overread,
- •9.Willing and able to comply with all protocol requirements and procedures,
- •10.Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations),
- •11.In France, a subject will be eligible for inclusion in this study only if either affiliated to or a beneficiary of a social security category.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 54
- •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
- •Subjects meeting any of the following criteria must not be enrolled in the study:
- •1.Any additional missing exon for DMD that cannot be treated with GSK2402968,
- •2.Current or history of liver or renal disease or impairment,
- •3.Acute illness within 4 weeks of the first anticipated administration of study medication which may interfere with study assessments,
- •4.Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs within 6 months of the first administration of study medication, and idebenone or other forms of Coenzyme Q10 within 1 month of the first administration of study medication,
- •5.Current or anticipated participation in any investigational clinical studies,
- •6.Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test at screening,
- •7.Symptomatic cardiomyopathy. If subject has a left ventricular ejection fraction <45% at Screening, the investigator should discuss inclusion of subject in the study with the medical monitor,
- •8.Children in Care. The definition of a Child in Care is a child who has been placed under the control or protection of an agency, organisation, institution or entity by the courts, the government or a government body, acting in accordance with powers conferred on them by law or regulation. The definition of a child in care can include a child cared for by foster parents or living in a care home or institution, provided that the arrangement falls within the definition above. The definition of a child in care does not include a child who is adopted or has an appointed legal guardian.
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