Exploratory Study of NS-065/NCNP-01 in Duchenne muscular dystrophy
- Conditions
- Duchenne muscular dystrophy
- Registration Number
- JPRN-UMIN000010964
- Lead Sponsor
- ational Center of Neurology and Psychiatry
- Brief Summary
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
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up complete
- Sex
- Male
- Target Recruitment
- 10
Not provided
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Safety (adverse event and adverse drug reaction)
- Secondary Outcome Measures
Name Time Method 1. Expression of dystrophin protein 2. Detection of exon53 skipped mRNA of dystrophin 3. NS-065/NCNP-01 concentration of the blood plasma 4. NS-065/NCNP-01 concentration of the urine 5. Serum Creatine kinase concentration