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

Last updated: December 12, 2024
Sponsor: NS Pharma, Inc.
Overall Status: Active - Recruiting

Phase

1/2

Condition

Muscular Dystrophy

Treatment

NS-050/NCNP-03

Placebo

Clinical Study ID

NCT06053814
NS-050/NCNP-03-101
  • Ages 4-14
  • Male

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Male ≥ 4 years and <15 years of age;

  • Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping ofexon 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 toremain on a stable dose for the duration of the study.

Other inclusion criteria may apply.

Exclusion

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 priorto first dose of study drug;

  • Currently taking another investigational drug or has taken another investigationaldrug 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 duringthe study duration;

  • Having taken any gene therapy.

Other exclusion criteria may apply.

Study Design

Total Participants: 20
Treatment Group(s): 2
Primary Treatment: NS-050/NCNP-03
Phase: 1/2
Study Start date:
September 18, 2024
Estimated Completion Date:
May 31, 2027

Connect with a study center

  • National Hospital Organization Nagara Medical Center

    Nagara, Gifu 502-8558
    Japan

    Active - Recruiting

  • Hyogo Medical University Hospital

    Nishinomiya, Hyogo 663-8501
    Japan

    Active - Recruiting

  • NHO Osaka Toneyama Medical Center

    Toyonaka, Osaka 560-8552
    Japan

    Active - Recruiting

  • National Center of Neurology and Psychiatry

    Kodaira, Tokyo 187-8551
    Japan

    Active - Recruiting

  • Children's Hospital Los Angeles

    Los Angeles, California 90027
    United States

    Site Not Available

  • Children's Hospital Colorado

    Aurora, Colorado 80011
    United States

    Active - Recruiting

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • University of Kansas Medical Center

    Kansas City, Kansas 66103
    United States

    Active - Recruiting

  • Shriners Children's Portland

    Portland, Oregon 97239
    United States

    Site Not Available

  • Shriners Hospital for Children

    Portland, Oregon 97239
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania 15213
    United States

    Site Not Available

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