NS-050/NCNP-03 in Boys With DMD (Meteor50)

April 20, 2026 updated by: NS Pharma, Inc.

A Phase 1/2, First in Human, Multiple-dose, 2-part Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-050/NCNP-03 in Boys With Duchenne Muscular Dystrophy (DMD)

This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 2
  • Phase 1

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

  • 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
    • Gifu
      • Nagara, Gifu, Japan, 502-8558
        • National Hospital Organization Nagara Medical Center
    • Hyōgo
      • Nishinomiya, Hyōgo, Japan, 663-8501
        • Hyogo Medical University Hospital
    • Miyagi
      • Sendai, Miyagi, Japan, 989-3126
        • Miyagi Children's Hospital
    • Osaka
      • Toyonaka, Osaka, Japan, 560-8552
        • NHO Osaka Toneyama Medical Center
    • Tokyo
      • Kodaira, Tokyo, Japan, 187-8551
        • National Center of Neurology and Psychiatry
  • South Korea
      • Seoul, South Korea
        • Seoul National University Hospital
    • Gyeongsangnam-do
      • Yangsan, Gyeongsangnam-do, South Korea
        • Pusan National University Yangsan Hospital
  • Turkey (Türkiye)
      • Ankara, Turkey (Türkiye)
        • Ankara Bilkent City Hospital
      • Istanbul, Turkey (Türkiye)
        • Istanbul University
      • Istanbul, Turkey (Türkiye)
        • Yeditepe University Kosuyolu Hospital
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
    • Colorado
      • Aurora, Colorado, United States, 80011
        • Children's Hospital Colorado
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Kansas
      • Kansas City, Kansas, United States, 66103
        • University of Kansas Medical Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh Medical Center

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 <16 years of age;
  • Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame;
  • Able to walk independently without assistive devices;
  • Able 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:

  • Evidence of symptomatic cardiomyopathy;
  • Current or previous treatment with anabolic steroids (e.g., oxendolone, oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug;
  • Currently taking another investigational drug or has taken another investigational drug within 3 months prior to the first dose of study drug;
  • Surgery within the 3 months prior to the first dose of study drug or planned during the study duration;
  • Having taken any gene therapy.

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Part 1: NS-050/NCNP-03
Participants will be randomized and receive NS-050/NCNP-03 intravenous (IV) infusions once weekly for 2 weeks at each of MAD levels (1.95, 5, 10, 20, 40, and 80 mg/kg).
Drug: NS-050/NCNP-03
NS-050/NCNP-03 solution for IV infusion.
Placebo Comparator: Part 1: Placebo
Participants will be randomized and receive NS-050/NCNP-03 placebo-matching IV infusions once weekly for 2 weeks at each of MAD levels.
Drug: Placebo
NS-050/NCNP-03 placebo-matching solution for IV infusion.
Experimental: Part 2: NS-050/NCNP-03
Participants will receive NS-050/NCNP-03 IV infusions once weekly for 24 weeks at the dosage selected by the Data and Safety Monitoring Board (DSMB) at the conclusion of Part 1.
Drug: NS-050/NCNP-03
NS-050/NCNP-03 solution for IV infusion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Part 1: Overall Summary of Treatment-emergent Adverse Events (TEAEs)
Time Frame: Baseline up to Week 24

TEAEs will be summarized both at the patient level for number of TEAEs, highest severity, relationship, action, and outcome, and at the TEAE level (summarizing events) by system organ class (SOC) and preferred term (PT) as well as severity, relationship, action, and outcome.

The most recent version of the Medical Dictionary for Regulatory Activities (MedDRA) will be used for coding TEAEs.
Baseline up to Week 24
Part 1: Area Under the Plasma Concentration Versus Time Curve (AUC) of NS-050/NCNP-03
Time Frame: Day 1 (1st dose) for each dose level
Blood samples will be collected at the designated time frame. Pharmacokinetic (PK) parameters of NS-050/NCNP-03 will be calculated using non-compartmental methods.
Day 1 (1st dose) for each dose level
Part 1: Amount of Drug Excreted in Urine of NS-050/NCNP-03
Time Frame: Day 1 (1st dose) for each dose level
Urine samples will be collected at the designated time frame. PK parameters of NS-050/NCNP-03 will be calculated using non-compartmental methods.
Day 1 (1st dose) for each dose level
Part 2: Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot)
Time Frame: Baseline, Week25
Baseline, Week25

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Part 2: Change from baseline in skeletal muscle dystrophin protein by mass spectrometry
Time Frame: Baseline, Week25
Baseline, Week25
Part 2: Change from baseline in skeletal muscle dystrophin protein levels by immunofluorescence staining
Time Frame: Baseline, Week25
Baseline, Week25
Part 2: Change from baseline in percentage of exon 50-skipped mRNA of skeletal muscle dystrophin
Time Frame: Baseline, Week25
Baseline, Week25
Part 2: 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
Part 2: Time to Stand (TTSTAND)
Time Frame: Baseline, Week13, Week25
Baseline, Week13, Week25
Part 2: Time to Run/Walk 10 Meters (TTRW)
Time Frame: Baseline, Week13, Week25
Baseline, Week13, Week25
Part 2: Time to Climb 4 Stairs (TTCLIMB)
Time Frame: Baseline, Week13, Week25
Baseline, Week13, Week25
Part 2: Total distance of 6 Minute Walk Test (6MWT)
Time Frame: Baseline, Week13, Week25
Baseline, Week13, Week25
Part 2: Muscle strength measured by Quantitative Muscle Testing (QMT)
Time Frame: Baseline, Week13, Week25
Baseline, Week13, Week25
Part 2: 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
Part 2: Grip/Pinch Strength
Time Frame: Baseline, Week13, Week25
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)

September 18, 2024

Primary Completion (Estimated)

May 1, 2027

Study Completion (Estimated)

March 1, 2028

Study Registration Dates

First Submitted

September 19, 2023

First Submitted That Met QC Criteria

September 19, 2023

First Posted (Actual)

September 26, 2023

Study Record Updates

Last Update Posted (Actual)

April 23, 2026

Last Update Submitted That Met QC Criteria

April 20, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NS-050/NCNP-03-101

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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