NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

• Status: Recruiting
• Trial end date: 2025-11-28
• Target enrollment: 0
• Participant gender: Male

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: NS Pharma, Inc.
• Collaborator: Nippon Shinyaku Co., Ltd.

Criteria

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 <7 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 taken any gene therapy or other exon-skipping oligonucleotide

• Other exclusion criteria may apply.