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Phase 2 Study of NS-089/NCNP-02 for Duchenne Muscular Dystrophy

This Phase 2 study is testing a drug called NS-089/NCNP-02 in boys with Duchenne Muscular Dystrophy (DMD) who have a specific genetic change (mutation) that could benefit from "exon 44 skipping." This means the drug aims to help the body make more of a protein called dystrophin, which is missing or faulty in DMD. The study is open to boys aged 4 to 14 years who can walk independently. Researchers will be looking at how safe the drug is and how the body processes it (pharmacokinetics). The study plans to enroll 20 participants, but its current status is unclear.

Study design
This is a Phase 2, open-label study, meaning both you and the researchers will know which treatment you are receiving. It involves 20 participants and is conducted across multiple centers.
What's involved
Participants will receive NS-089/NCNP-02 as a weekly intravenous (IV) infusion. The study has two parts, with treatment phases lasting 4 weeks at different dose levels and then a 24-week treatment phase.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed through study completion, which includes a follow-up phone call for Part 2 of the study.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05996003

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

Recruiting
PHASE2Ages 4–14InterventionalTreatment
NS Pharma, Inc.
~20 participants
Updated 2026-03-06 on ClinicalTrials.gov
What's tested:NS-089/NCNP-02

At a glance

Recruiting sites
12 of 25 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Adverse Event and Adverse Drug Reaction
Measured over through study completion, up to follow-up phone call for Part 2
+9 more outcomes measured

Conditions

Duchenne Muscular Dystrophy
Exon 44
DMD

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Duchenne Muscular DystrophyDMD Gene

Where it's being run

25 sites across 22 states
Turkey (Türkiye)3
Pennsylvania2
Colorado1
Florida1
Georgia1
Illinois1
Kansas1
Ohio1

Who to contact

Trial info
[email protected]
1-866-677-6276
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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.

Exclusion

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

What this trial measures

  • Adverse Event and Adverse Drug Reactionthrough study completion, up to follow-up phone call for Part 2
  • Plasma pharmacokinetic (PK) parametersDay1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Maximum plasma concentration (Cmax) of NS-089/NCNP-02
  • Plasma pharmacokinetic (PK) parametersDay1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Time of the maximum plasma concentration (Tmax) of NS-089/NCNP-02
  • Plasma pharmacokinetic (PK) parametersDay1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Terminal half-life (T1/2) of NS-089/NCNP-02
  • Plasma pharmacokinetic (PK) parametersDay1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to the last time point (AUC0-t) of NS-089/NCNP-02
  • Plasma pharmacokinetic (PK) parametersDay1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to infinity (AUC0-∞) of NS-089/NCNP-02
  • Plasma pharmacokinetic (PK) parameters[Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Total body clearance (CLtot) of NS-089/NCNP-02
  • Plasma pharmacokinetic (PK) parametersDay1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] The volume in the terminal state (Vz) of NS-089/NCNP-02
  • Urine pharmacokinetic parametersDay1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Urinary excretion of NS-089/NCNP-02
  • Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot).Baseline, Week25