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

Actively RecruitingPhase 2Exon Skipping

Age: 4–14 years
Phase: Phase 2
Sponsor: NS Pharma
Therapeutic Approach: Exon Skipping
Variant Requirement: Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame.
Eligible Sex: Male
Ambulation: Ambulatory
Study Start (Actual): 2024-02-22
Primary Completion (Estimated): 2026-09-11
Study Completion (Estimated): 2026-09-11
Enrollment (Estimated): 20
Countries: United StatesAustraliaCanadaJapanNew ZealandSouth KoreaTurkey

Male ≥ 4 years and <15 years of age
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.

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.

This section provides contact details for people who can answer questions about joining this study

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

Study Requirements and Criteria