Status: Recruiting
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \<15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly. The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.
Participants needed: 20
Trial details
Phase: Phase 2Age: 4-14Biological sex: MaleType: InterventionalSponsor: NS Pharma, Inc.Updated: Mar 6, 2026Locations: 25
Eligibility criteria
Male ≥ 4 years and <15 years of age [+5]
Has a body weight of <20 kg at the time of informed consent (applies to particip... [+7]