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NCT05996003PHASE2Recruiting

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

NS Pharma, Inc.

Start Date

2/22/2024

Completion Date

9/11/2026

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 Criteria

Age Range: 4 years to 14 years

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.

Interventions

DRUG

NS-089/NCNP-02

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Conditions

Duchenne Muscular DystrophyExon 44DMD

Locations

Children's Hospital Colorado

Aurora, Colorado 80045

United States

Rare Disease Research, LLC - FL

Kissimmee, Florida 34746

United States

Rare Disease Research

Atlanta, Georgia 30329

United States

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois 60611

United States

University of Kansas Medical Center (KUMC)

Kansas City, Kansas 66160

United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio 45229

United States

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania 19104

United States

University of Pittsburgh School of Medicine

Pittsburgh, Pennsylvania 15224

United States

UT Southwestern/Children's Health

Dallas, Texas 75207

United States

Virginia Commonwealth University Health System

Richmond, Virginia 23298

United States

Perth Children's Hospital

Nedlands, Western Australia 6009

Australia

Alberta Children's Hospital

Calgary, Alberta

Canada

British Columbia Children's Hospital

Vancouver, British Columbia

Canada

London Health Sciences Centre

London, Ontario

Canada

Fukui Prefectural Hospital

Fukui-shi, Fukui 910-8526

Japan

National Hospital Organization Nagara Medical Center

Nagara, Gifu-shi, Gifu 502-8558

Japan

NHO Osaka Toneyama Medical Center

Toyonaka, Osaka 560-8552

Japan

Shiga General Hospital

Moriyama-shi, Shiga 524-8524

Japan

National Center of Neurology and Psychiatry

Kodaira, Tokyo 187-8551

Japan

Starship Children's Hospital

Auckland, 1023

New Zealand

Pusan National University Yangsan Hospital

Yangsan, Gyeongsangnam

South Korea

Seoul National University Hospital

Seoul,

South Korea

Ankara Bilkent City Hospital

Ankara, 06800

Turkey (Türkiye)

Yeditepe University Kosuyolu Hospital

Istanbul, 34718

Turkey (Türkiye)

S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital

Izmir, 11794

Turkey (Türkiye)