NL-OMON34487已完成2 期
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 - GSK2402968 in subjects with Duchenne muscular dystrophy
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 17（—）
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. Results of 6MWD and rise-from-floor tests must be reproducible (within 20% for
- •each test) between Screening Visits 1 and 2
- •8. 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
- •9. 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
- •10. Subjects must be willing to use adequate contraception (condoms or abstinence) for the duration of the study and for at least 5 months after the last dose of study drug
- •11. Willing and able to comply with all protocol requirements and procedures
- •12. Able to give informed assent and/or consent in writing signed by the subject
- •and/or parent(s)/legal guardian (according to local regulations)
- •13. 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
排除标准
- •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.
研究者
相似试验
进行中（未招募）
1 期
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 dystrophyMedDRA version: 14.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersDuchenne Muscular DystrophyEUCTR2010-018412-32-BEGlaxoSmithKline Research and Development LTD54
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 dystrophyDuchenne Muscular DystrophyMedDRA version: 14.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2010-018412-32-GBGlaxoSmithKline Research and Development LTD54
进行中（未招募）
1 期
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 dystrophyMedDRA version: 12.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophyDuchenne Muscular DystrophyEUCTR2010-018412-32-FRGlaxoSmithKline Research and Development LTD53
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 dystrophyDuchenne Muscular DystrophyMedDRA version: 12.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophyEUCTR2010-018412-32-NLGlaxoSmithKline Research and Development LTD54
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 dystrophyMedDRA version: 14.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersDuchenne Muscular DystrophyEUCTR2010-018412-32-Outside-EU/EEAGlaxoSmithKline Research and Development LTD54