Uken

Researchers are evaluating NS-089NCNP-02 in a Phase 2, open-label study involving ambulant boys aged 4 to less than 15 years with Duchenne Muscular Dystrophy DMD caused by mutations suitable for exon 44 skipping. The study aims to assess the safety, tolerability, pharmacokinetics, and effects of the drug on muscle dystrophin levels and physical function. This research is sponsored by NS Pharma, Inc. and involves two parts with distinct participant cohorts. The study includes two parts Part 1 involves six participants Cohort 1 receiving escalating doses of NS-089NCNP-02 by weekly intravenous infusion over a 4-week treatment phase per dose level. Part 2 involves both Cohort 1 and 14 additional participants Cohort 2 receiving a selected dose of NS-089NCNP-02 once weekly for 24 weeks. The drug is administered as a solution for infusion, and dosing in Part 2 is based on the maximum tolerated dose determined in Part 1. Participants will undergo assessments at baseline, during treatment, and follow-up visits including pharmacokinetic blood and urine tests, muscle biopsies to measure dystrophin protein and mRNA, and physical function tests such as the North Star Ambulatory Assessment, timed walking and climbing tasks, muscle strength tests, and grip and pinch strength. Safety monitoring includes tracking adverse events throughout the study and follow-up. The overall participation lasts through the treatment phases and a follow-up phone call after Part 2 completion.