Exploratory Study of NS-065/NCNP-01 in Duchenne muscular dystrophy - Exploratory Study of NS-065/NCNP-01

ational Center of Neurology and Psychiatry0 个研究点目标入组 10 人开始时间: 2013年6月17日最近更新: 2023年10月17日

适应症

试验速览

阶段: 1 期
状态: 已完成
发起方
入组人数: 10

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

Komaki.H, Nagata.T, Saito.T, Masuda.S, Takeshita.E, Sasaki.M, Tachimori.H, Nakamura.H, Aoki.Y, Takeda.S Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy. Sci Transl Med. 2018, 10(437) https://stm.sciencemag.org/content/10/437/eaan0713.short

研究设计

研究类型: Interventional

入排标准

年龄范围: 5years-old 至 18years-old（—）
性别: Male

入选标准

未提供

排除标准

•Subject with Duchenne muscular dystrophy meeting any of the following criteria must not be enrolled in the study: 1)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. 2) A forced vital capacity (FVC) < 50% of predicted. 3) A left ventricular ejection fraction (EF) < 40% or fractional shortening (FS) < 25% based on echocardiogram (ECHO). 4) Surgery within the last 3 months prior to the first anticipated administration of study medication or planned for anytime during the duration of the study. 5) Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at screening. 6) Current diagnosis of any immune deficiency or autoimmune disease. 7) Current diagnosis of any active or uncontrolled infection, cardiomyopathy, or liver or renal disease. 8) Use of any other investigational agents and/or experimental agents within 3 months prior to the first anticipated administration of study medication. 9) History of any severe drug allergy. 10) Unable to give informed consent about using adequate contraception from the first administration until at least 6 months after the last dose of study medication, by parent(s) or legal guardian. 11) Subject considered by the investigator (or sub-investigator), for any reason, to be an unsuitable candidate for the study.

研究者

发起方

ational Center of Neurology and Psychiatry

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