A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)

Trial ID: NCT05996003

Interventional - Phase 2 - Active, not recruiting

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.

Conditions

• Duchenne Muscular Dystrophy
• Exon 44
• DMD

Interventions

• NS-089/NCNP-02

Eligibility

Inclusion criteria

• Male ≥ 4 years and \<15 years of age
• Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame
• Able to walk independently without assistive devices
• Ability to complete the TTSTAND without assistance in \<20 seconds
• Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.
• Other inclusion criteria may apply.

Exclusion criteria

• Has a body weight of \<20 kg at the time of informed consent (applies to participants screening for Part 1 only)
• Evidence of symptomatic cardiomyopathy
• Current or previous treatment with anabolic steroids (e.g., oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug
• Current or previous treatment with any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer
• Surgery within the 3 months prior to the first dose of study drug or planned during the study duration
• Previously treated in an interventional study of NS-089/NCNP-02
• Having received exon skipping oligonucleotide within 1 year prior to the first dose of IP
• Other exclusion criteria may apply.

Sponsor

NS Pharma, Inc., Nippon Shinyaku Co., Ltd.

Source: ClinicalTrials.gov

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