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

September 10, 2026 updated by: NS Pharma, Inc.

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Queensland
      • South Brisbane, Queensland, Australia, 4101
        • Queensland Children's Hospital
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Perth Children's Hospital
  • Canada
    • Alberta
      • Calgary, Alberta, Canada
        • Alberta Children's Hospital
    • British Columbia
      • Vancouver, British Columbia, Canada
        • British Columbia Children's Hospital
    • Ontario
      • London, Ontario, Canada
        • London Health Sciences Centre
  • Japan
    • Fukui
      • Fukui-shi, Fukui, Japan, 910-8526
        • Fukui Prefectural Hospital
    • Gifu-shi, Gifu
      • Nagara, Gifu-shi, Gifu, Japan, 502-8558
        • National Hospital Organization Nagara Medical Center
    • Osaka
      • Toyonaka, Osaka, Japan, 560-8552
        • NHO Osaka Toneyama Medical Center
    • Shiga
      • Moriyama-shi, Shiga, Japan, 524-8524
        • Shiga General Hospital
    • Tokyo
      • Kodaira, Tokyo, Japan, 187-8551
        • National Center of Neurology and Psychiatry
  • New Zealand
      • Auckland, New Zealand, 1023
        • Starship Children's Hospital
  • South Korea
      • Seoul, South Korea
        • Seoul National University Hospital
    • Gyeonggi-do
      • Seongnam-si, Gyeonggi-do, South Korea, 13620
        • Seoul National University Bundang Hospital
    • Gyeongsangnam
      • Yangsan, Gyeongsangnam, South Korea
        • Pusan National University Yangsan Hospital
  • Turkey (Türkiye)
      • Ankara, Turkey (Türkiye), 06800
        • Ankara Bilkent City Hospital
      • Istanbul, Turkey (Türkiye), 34718
        • Yeditepe University Kosuyolu Hospital
      • Istanbul, Turkey (Türkiye), 34093
        • Istanbul University- Istanbul Faculty of Medicine
      • Izmir, Turkey (Türkiye), 11794
        • S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital
  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Rare Disease Research
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann and Robert H. Lurie Children's Hospital of Chicago
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center (KUMC)
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • New York
      • New York, New York, United States, 10032
        • Columbia University Pediatric Neuromuscular Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia (CHOP)
      • Pittsburgh, Pennsylvania, United States, 15224
        • University of Pittsburgh School of Medicine
    • Texas
      • Dallas, Texas, United States, 75207
        • UT Southwestern/Children's Health
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University Health System

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: NS-089/NCNP-02

Experimental: NS-089/NCNP-02

NS-089/NCNP-02 solution for infusion (Cohort 1)

NS-089/NCNP-02 solution for infusion (Cohort 2)
Drug: 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

Other Names:
  • Brogidirsen

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Adverse Event and Adverse Drug Reaction
Time Frame: through study completion, up to follow-up phone call for Part 2
through study completion, up to follow-up phone call for Part 2
Plasma pharmacokinetic (PK) parameters
Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Maximum plasma concentration (Cmax) of NS-089/NCNP-02
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
Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Terminal half-life (T1/2) of NS-089/NCNP-02
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
Time Frame: 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
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
Time Frame: 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
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
Urine pharmacokinetic parameters
Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Urinary excretion of NS-089/NCNP-02
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).
Time Frame: Baseline, Week25
Baseline, Week25
Plasma pharmacokinetic (PK) parameters
Time Frame: 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
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
Time Frame: 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
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
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
Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Total body clearance (CLtot) of NS-089/NCNP-02

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in skeletal muscle dystrophin protein by mass spectrometry.
Time Frame: Baseline, Week25
Baseline, Week25
Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining.
Time Frame: Baseline, Week25
Baseline, Week25
Change from baseline in percentage of exon 44-skipped mRNA of skeletal muscle dystrophin
Time Frame: Baseline, Week25
Baseline, Week25
North Star Ambulatory Assessment (NSAA) score
Time Frame: Baseline, Week13, Week25
The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.
Baseline, Week13, Week25
Time to Run/Walk 10 Meters (TTRW)
Time Frame: Baseline, Week13, Week25
Baseline, Week13, Week25
Time to Stand (TTSTAND)
Time Frame: Baseline, Week13, Week25
Baseline, Week13, Week25
Total distance of 6 Minute Walk Test (6MWT)
Time Frame: Baseline, Week13, Week25
Baseline, Week13, Week25
Time to Climb 4 Stairs (TTCLIMB)
Time Frame: Baseline, Week13, Week25
Baseline, Week13, Week25
Muscle strength measured by Quantitative Muscle Testing (QMT)
Time Frame: Baseline, Week13, Week25
Baseline, Week13, Week25
Grip and pinch strength
Time Frame: Baseline, Week13, Week25
Baseline, Week13, Week25
Performance of Upper Limb (PUL) 2.0. score
Time Frame: Baseline, Week13, Week25
The PUL 2.0 provides both a total score and sub-scores for the 3 domains (shoulder, middle, and distal) that in DMD are progressively involved with a proximal to distal gradient. The PUL includes 22 items with an entry item to define the starting functional level. The 22 items are subdivided into the high-level shoulder dimension (6 items), middle level elbow dimension (9 items), and distal wrist and hand dimension (7 items). For weaker patients, a low score on the entry item (0-2) means high level items do not need to be performed. Scoring options vary across the scale between 0-1 and 0-2 according to performance. Each dimension can be scored separately with a maximum score of 12 for the high-level shoulder dimension, 17 for the middle level elbow dimension, and 13 for the distal wrist and hand dimension. A total score can be achieved by adding the 3 level scores (maximum total score of 42).
Baseline, Week13, Week25

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

NS Pharma, Inc.

Collaborators

Nippon Shinyaku Co., Ltd.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 22, 2024

Primary Completion (Estimated)

February 26, 2027

Study Completion (Estimated)

February 26, 2027

Study Registration Dates

First Submitted

July 18, 2023

First Submitted That Met QC Criteria

August 10, 2023

First Posted (Actual)

August 16, 2023

Study Record Updates

Last Update Posted (Actual)

September 15, 2026

Last Update Submitted That Met QC Criteria

September 10, 2026

Last Verified

September 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NS-089/NCNP-02-201

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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