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

Recruiting now · Phase 2

Conditions studied: Duchenne Muscular Dystrophy, Exon 44, DMD

In brief

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.

Key facts

Study ID

NCT05996003

Run by

NS Pharma, Inc.

People needed

20

Starts

2024-02-22

Expected to finish

2026-09-11

Last updated by the study team

2026-03-06

Who can join

Age: 4 and older, up to 14. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• London Health Sciences Centre — London, Ontario, Canada (enrolling)
• University of Kansas Medical Center (KUMC) — Kansas City, Kansas, United States (enrolling)
• National Hospital Organization Nagara Medical Center — Nagara, Gifu-shi, Gifu, Japan (enrolling)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
• The Children's Hospital of Philadelphia (CHOP) — Philadelphia, Pennsylvania, United States (enrolling)
• University of Pittsburgh School of Medicine — Pittsburgh, Pennsylvania, United States (enrolling)
• UT Southwestern/Children's Health — Dallas, Texas, United States (enrolling)
• Virginia Commonwealth University Health System — Richmond, Virginia, United States (enrolling)
• Rare Disease Research, LLC - FL — Kissimmee, Florida, United States (enrolling)
• Rare Disease Research — Atlanta, Georgia, United States (enrolling)
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
• Children's Hospital Colorado — Aurora, Colorado, United States (enrolling)
• Yeditepe University Kosuyolu Hospital — Istanbul, Turkey (Türkiye)
• S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital — Izmir, Turkey (Türkiye)
• Perth Children's Hospital — Nedlands, Western Australia, Australia
• Alberta Children's Hospital — Calgary, Alberta, Canada
• British Columbia Children's Hospital — Vancouver, British Columbia, Canada
• Fukui Prefectural Hospital — Fukui-shi, Fukui, 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
• Pusan National University Yangsan Hospital — Yangsan, Gyeongsangnam, South Korea
• Seoul National University Hospital — Seoul, South Korea
• Ankara Bilkent City Hospital — Ankara, Turkey (Türkiye)

Full record on ClinicalTrials.gov

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