Uken

Researchers are evaluating NS-089/NCNP-02, a drug given by weekly intravenous infusion, in ambulatory boys aged 4 to under 15 years with Duchenne Muscular Dystrophy (DMD) caused by mutations that can be treated by skipping exon 44. This Phase 2, open-label, multi-center study aims to assess the drug's safety, tolerability, effectiveness, and how the body processes it in this specific group of participants. The study has two parts. Part 1 involves six boys (Cohort 1) who receive three dose levels of NS-089/NCNP-02 weekly over 4 weeks each. Part 2 includes Cohort 1 and an additional 14 boys (Cohort 2) who receive the selected maximum tolerated dose (MTD) weekly for 24 weeks. The treatment is given by intravenous infusion, and dosing is adjusted based on Part 1 results. Participants will be monitored through various assessments including blood and urine tests to study pharmacokinetics, muscle biopsies to measure changes in dystrophin protein, and safety evaluations for adverse events. These measurements are taken at baseline, during dosing, and at follow-up times up to 24 weeks, with additional phone follow-ups to monitor ongoing safety.