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

Phase 1

• Conditions: Duchenne muscular dystrophy

• Registration Number: JPRN-UMIN000038505
• Lead Sponsor: ational Center of Neurology and Psychiatry

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-11-06
• Study Completion: 2021-12-31
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete: follow-up complete
• Sex: Male
• Target Recruitment: 6

Inclusion Criteria

Not provided

Exclusion Criteria

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.

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 exon 44-skipped mRNA of dystrophin 3. Time to Stand Test (TTSTAND) 4. Time to Run/Walk 10 Meters test (TTRW) 5. Six-Minute Walk Test (6MWT) and Two-Minute Walk Test (2MWT) 6. Timed Up & Go (TUG) test 7. Performance of Upper Limb (PUL) test 8. NS-089/NCNP-02 concentration of the blood plasma 9. NS-089/NCNP-02 concentration of the urine 10. Serum Creatine kinase concentration