Home › Trials › NCT05996003

Treatment for boys with Duchenne Muscular Dystrophy using NS-089/NCNP-02

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

PHASE2 · NS Pharma, Inc. · NCT05996003

This study is testing a new treatment called NS-089/NCNP-02 to see if it can help boys aged 4 to under 15 with Duchenne Muscular Dystrophy who can walk on their own.

Quick facts

PhasePHASE2
Study typeInterventional
Enrollment20 (estimated)
Ages4 Years to 14 Years
SexMale
SponsorNS Pharma, Inc. (industry)
Locations25 sites (Aurora, Colorado and 24 other locations)
Trial IDNCT05996003 on ClinicalTrials.gov

What this trial studies

This Phase 2, open-label, multi-center study evaluates the safety and efficacy of NS-089/NCNP-02, administered via weekly IV infusion, in ambulant boys aged 4 to under 15 years with Duchenne Muscular Dystrophy (DMD) due to specific mutations. The study is divided into two parts, with a total of 20 participants across two cohorts. Participants must be able to walk independently and meet specific health criteria to be eligible for the treatment.

Who should consider this trial

Good fit: Ideal candidates are male children aged 4 to under 15 years with specific DMD mutations that allow for exon 44 skipping.

Not a fit: Patients with symptomatic cardiomyopathy or those who have recently used certain anabolic steroids or investigational drugs may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve muscle function and quality of life for boys with Duchenne Muscular Dystrophy.

How similar studies have performed: Other studies targeting similar mutations in DMD have shown promise, indicating potential for success with this approach.

Eligibility criteria

Show full inclusion / exclusion 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 \<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.

Where this trial is running

Aurora, Colorado and 24 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Duchenne Muscular Dystrophy, Exon 44, DMD, Exon 44 Skipping, Brogidirsen

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.