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Clinical Trials in South Korea / NCT05996003
Active, not recruiting Phase 2

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

NCT05996003 · tracked via the Priya Life Science South Korea tracker
Sponsor
NS Pharma, Inc
Phase
Phase 2
Started
2024-02-22
Last updated
2026-09-15

Condition(s) studied

Duchenne Muscular DystrophyExon 44DMD

Investigational drug(s) / intervention(s)

NS-089/NCNP-02 →

NS-089/NCNP-02: 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

Study 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.

Eligibility

Sex
MALE
Min age
4 Years
Max age
14 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Adverse Event and Adverse Drug Reaction — through study completion, up to follow-up phone call for Part 2
  • Plasma pharmacokinetic (PK) parameters — Day1, 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) parameters — Day1, 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) parameters — Day1, 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) parameters — Day1, 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) parameters — Day1, 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 — 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) parameters — Day1, 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 parameters — Day1, 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

Trial sites (29)

FacilityCityRegionStatus
Children's Hospital Colorado Aurora Colorado
Rare Disease Research Atlanta Georgia
Ann and Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
University of Kansas Medical Center (KUMC) Kansas City Kansas
Boston Children's Hospital Boston Massachusetts
Columbia University Pediatric Neuromuscular Center New York New York
Cincinnati Children's Hospital Medical Center Cincinnati Ohio
The Children's Hospital of Philadelphia (CHOP) Philadelphia Pennsylvania
University of Pittsburgh School of Medicine Pittsburgh Pennsylvania
UT Southwestern/Children's Health Dallas Texas
Virginia Commonwealth University Health System Richmond Virginia
Queensland Children's Hospital South Brisbane Queensland
Perth Children's Hospital Nedlands Western Australia
Alberta Children's Hospital Calgary Alberta
British Columbia Children's Hospital Vancouver British Columbia
London Health Sciences Centre London Ontario
Fukui Prefectural Hospital Fukui-shi Fukui
National Hospital Organization Nagara Medical Center Nagara Gifu-shi, Gifu
NHO Osaka Toneyama Medical Center Toyonaka Osaka
Shiga General Hospital Moriyama-shi Shiga
National Center of Neurology and Psychiatry Kodaira Tokyo
Starship Children's Hospital Auckland New Zealand
Seoul National University Bundang Hospital Seongnam-si Gyeonggi-do
Pusan National University Yangsan Hospital Yangsan Gyeongsangnam
Seoul National University Hospital Seoul South Korea
Ankara Bilkent City Hospital Ankara Turkey (Türkiye)
Istanbul University- Istanbul Faculty of Medicine Istanbul Turkey (Türkiye)
Yeditepe University Kosuyolu Hospital Istanbul Turkey (Türkiye)
S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital Izmir Turkey (Türkiye)

More NS Pharma, Inc. trials in South Korea

NS-050/NCNP-03 in Boys With DMD (Meteor50) Active, not recruiting

Other trials for the same condition

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) Active, not recruiting
NS-050/NCNP-03 in Boys With DMD (Meteor50) Active, not recruiting
ML-Based Multi-Sensor Fall Risk Screening in DMD Recruiting
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05996003 on ClinicalTrials.gov ↗ ← All trials in South Korea