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RECRUITINGPhase 2INTERVENTIONAL

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

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - 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. Who Should NOT Join This Trial: - Has a body weight of \<20 kg at the time of willing to sign a consent form (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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

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

Locations (20)

Children's Hospital Colorado
Aurora, Colorado, United States
Rare Disease Research, LLC - FL
Kissimmee, Florida, United States
Rare Disease Research
Atlanta, Georgia, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, United States
UT Southwestern/Children's Health
Dallas, Texas, United States
Virginia Commonwealth University Health System
Richmond, Virginia, United States
Perth Children's Hospital
Nedlands, Western Australia, 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, Japan
National Hospital Organization Nagara Medical Center
Nagara, Gifu-shi, Gifu, Japan
NHO Osaka Toneyama Medical Center
Toyonaka, Osaka, Japan
Shiga General Hospital
Moriyama-shi, Shiga, Japan
National Center of Neurology and Psychiatry
Kodaira, Tokyo, Japan
Starship Children's Hospital
Auckland, New Zealand