Exploratory Study of NS-089/NCNP-02 in Duchenne muscular dystrophy - Exploratory Study of NS-089/NCNP-02

ational Center of Neurology and Psychiatry0 个研究点目标入组 6 人开始时间: 2019年11月6日最近更新: 2023年10月17日

适应症

试验速览

暂无简介。

•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. Continuous use of artificial respirator (except for use of NPPV while sleeping) 4. A left ventricular ejection fraction (EF) < 40% or fractional shortening (FS) < 25% based on echocardiogram (ECHO). 5. 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 2. 6. Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at screening. 7. Current diagnosis of any immune deficiency or autoimmune disease. 8. Current diagnosis of any active or uncontrolled infection, cardiomyopathy, or liver or renal disease. 9. Use of any other investigational agents and/or experimental agents within 3 months prior to the first anticipated administration of study medication. 10. History of any severe drug allergy.

发起方

ational Center of Neurology and Psychiatry