EUCTR2010-024566-22-FR进行中（未招募）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 - N/A
GlaxoSmithKline Research and Development LTD0 个研究点目标入组 32 人开始时间: 2011年2月22日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 32
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
- •Subjects eligible for enrolment in the study must meet all of the following criteria:
- •1. 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 GSK2402968.
- •2. Age 9 years old or greater at Screening;
- •4. Non-ambulant (at least 1 year in a wheelchair) within the last 4 years;
- •5. Life expectancy at least three years;
- •6. Willingness and ability to comply with all protocol requirements and procedures;
- •7. 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;
- •8. 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;
- •9. Informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations).
- •10 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:
- •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 mutation (such as an additional missing exon for DMD) that cannot
- •be treated with GSK2402968;
- •2. Current or history of liver or renal disease;
- •3. Acute illness within 4 weeks of anticipated administration of study medication,
- •which may interfere with study assessments;
- •4. Daytime ventilator-dependency (except for daytime naps);
- •5. 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 study
- •medication.
- •6. Start of glucocorticosteroids within 6 months or non-stable use of
- •glucocorticosteroids within 3 months of the anticipated first administration of study
- •medication;
- •7. Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or
- •human immunodeficiency virus (HIV) test at Screening;
- •8. Symptomatic cardiomyopathy;
- •9. Use of alcohol from Screening through to the 1 month Follow-up visit (Day 29-35,
- •inclusive);
- •10. Any Child in Care. (The definition of a Child in Care is a child who has been placed
- •under the control or protection of an agency, organization, 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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