NCT04129294已完成1 期
Exploratory Study of NS-089/NCNP-02 in Duchenne Muscular Dystrophy
National Center of Neurology and Psychiatry, Japan1 个研究点 分布在 1 个国家目标入组 6 人开始时间: 2019年12月2日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Adverse event and adverse drug reaction [Safety and Tolerability]
研究概览
简要总结
This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-089/NCNP-02 in subjects diagnosed with Duchenne muscular dystrophy (DMD), and to determine the dosage for subsequent studies.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 17 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Has an out of frame deletion(s) that could be corrected by skipping exon 44 as confirmed by any of methodology at the time of visit
- •If not confirmed by any of methodology that evaluates the relative copy number of all exons (i.e. MLPA etc), must be confirmed through these techniques by the time of visit
- •DNA sequencing of exon 44 confirms that no DNA polymorphisms occur that could compromise duplex formation between NS-089/NCNP-02 and pre-mRNA.
- •Male and >= 8 years and < 17 years of age at the time of obtaining informed consent and/or assent. Subjects aged >= 4 years and < 8 years can be enrolled according to the circumstances.
- •Able to give informed consent in writing signed by parent(s) or legal guardian who is able to understand all of the study procedure requirements. If applicable, able to give informed assent in writing signed by the subject.
- •Life expectancy of at least 1 year
- •Able to ambulate. Non-ambulant subject can be enrolled according to the circumstances.
- •Have intact muscles, which have adequate quality for biopsy. (No lacks or severe atrophy of biceps brachii or tibialis anterior muscle)
- •QTc <450 msec (based on 12-lead ECGs), or <480 msec for subject with Bundle Branch Block.
- •Glucocorticoid-naive patients, or patients who have used systemic glucocorticoids for at least 6 months prior to enrollment in this study with no dose changes for at least 3 months prior to enrollment.
排除标准
- •Has participated in other pharmacological clinical trial that might recover dystrophin protein by the readthrough or the exon-skipping therapy, and/or upregulate the dystrophin-associated proteins such as utrophin.
- •A forced vital capacity (FVC) < 50% of predicted.
- •Continuous use of artificial respirator (except for use of NPPV while sleeping)
- •A left ventricular ejection fraction (EF) < 40% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
- •Surgery within the last 3 months prior to the first anticipated administration of study medication or planned for anytime between visit 1 of Part 1 and the last visit of Part
- •Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at screening.
- •Current diagnosis of any immune deficiency or autoimmune disease.
- •Current diagnosis of any active or uncontrolled infection, cardiomyopathy, or liver or renal disease.
- •Use of any other investigational agents and/or experimental agents within 3 months prior to the first anticipated administration of study medication.
- •History of any severe drug allergy.
研究组 & 干预措施
NS-089/NCNP-02
Experimental
NS-089/NCNP-02
干预措施: NS-089/NCNP-02 (Drug)
结局指标
主要结局
Adverse event and adverse drug reaction [Safety and Tolerability]
时间窗: At the end of Part 2 (24 weeks treatment period and 12 weeks follow up period)
adverse event and adverse drug reaction
次要结局
- NS-089/NCNP-02 concentration of the blood plasma(At the end of Part 2 (24 weeks treatment period and 12 weeks follow up period))
- TTSTAND(At the end of the treatment period (24 weeks) of Part 2)
- TTRW(At the end of the treatment period (24 weeks) of Part 2)
- PUL(At the end of the treatment period (24 weeks) of Part 2)
- Expression of dystrophin protein(At the end of the treatment period (24 weeks) of Part 2)
- NSAA(At the end of the treatment period (24 weeks) of Part 2)
- 6MWT and 2MWT(At the end of the treatment period (24 weeks) of Part 2)
- TUG(At the end of the treatment period (24 weeks) of Part 2)
- Detection of exon 44-skipped mRNA of dystrophin in muscle tissue(At the end of the treatment period (24 weeks) of Part 2)
- Serum Creatine kinase concentration(At the end of Part 2 (24 weeks treatment period and 12 weeks follow up period))
研究者
Hirofumi Komaki
Director
National Center of Neurology and Psychiatry, Japan
研究点 (1)
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