NIPH Clinical Trials Search

A Global Phase 2 study of NS-089/NCNP-02 in Boys with Duchenne Muscular Dystrophy (DMD)

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	Duchenne muscular dystrophy
Date of first enrollment	28/05/2025
Target sample size	20
Countries of recruitment	USA,Japan,Canada,Japan,South Korea,Japan,New Zealand,Japan,Australia,Japan,Turkey,Japan,UK,Japan
Study type	Interventional
Intervention(s)	Cohort 1: Part 1 Dose Level 1-3: a 4-week Treatment Phase at each treatment dose level Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1 Cohort 2: Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1

Outcome(s)

Primary Outcome	-Adverse Event and Adverse Drug Reaction -Plasma pharmacokinetic (PK) parameters -Urine pharmacokinetic parameters -Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot).
Secondary Outcome	-Biceps brachii muscle dystrophin protein; -Biceps brachii muscle dystrophin mRNA levels; -NSAA; -TTRW; -TTSTAND; -6MWT; -TTCLIMB; -QMT ; -Grip and Pinch strength; and -PUL 2.0.

Key inclusion & exclusion criteria

Age minimum	>= 4age old
Age maximum	< 15age old
Gender	Male
Include 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.
Exclude 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 -Participant has received exon skipping oligonucleotide therapy within 1 year prior to the first dose of IP -Other exclusion criteria may apply.