New hope for duchenne: experimental drug targets genetic glitch

NCT ID NCT05996003

Recruiting now Disease control Sponsor: NS Pharma, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests an experimental drug called NS-089/NCNP-02 (Brogidirsen) in 20 boys with Duchenne muscular dystrophy whose genetic mutation can be fixed by skipping exon 44. The drug is given as a weekly IV infusion and aims to help the body produce a working version of the dystrophin protein. Researchers will check safety, how the drug moves through the body, and whether it increases dystrophin levels in muscle.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: NS-089/NCNP-02 (Brogidirsen)
What this could lead to: If successful, this could lead to a treatment that helps boys with Duchenne muscular dystrophy produce a shorter but functional dystrophin protein, potentially slowing muscle decline.
What could go wrong: This is an early Phase 2 trial with only 20 participants, so results may not apply to all. The drug may not produce enough dystrophin to meaningfully change the disease course, and side effects are still being studied.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DMD DUCHENNE MUSCULAR DYSTROPHY EXON 44

Contacts and locations

Locations

Alberta Children's Hospital
NOT_YET_RECRUITING

Calgary, Alberta, Canada
Ankara Bilkent City Hospital
NOT_YET_RECRUITING

Ankara, 06800, Turkey (Türkiye)
Ann and Robert H. Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
British Columbia Children's Hospital
NOT_YET_RECRUITING

Vancouver, British Columbia, Canada
Children's Hospital Colorado
RECRUITING

Aurora, Colorado, 80045, United States
Cincinnati Children's Hospital Medical Center
RECRUITING

Cincinnati, Ohio, 45229, United States
Fukui Prefectural Hospital
NOT_YET_RECRUITING

Fukui-shi, Fukui, 910-8526, Japan
London Health Sciences Centre
RECRUITING

London, Ontario, Canada
NHO Osaka Toneyama Medical Center
NOT_YET_RECRUITING

Toyonaka, Osaka, 560-8552, Japan
National Center of Neurology and Psychiatry
NOT_YET_RECRUITING

Kodaira, Tokyo, 187-8551, Japan
National Hospital Organization Nagara Medical Center
RECRUITING

Nagara, Gifu-shi, Gifu, 502-8558, Japan
Perth Children's Hospital
NOT_YET_RECRUITING

Nedlands, Western Australia, 6009, Australia
Pusan National University Yangsan Hospital
NOT_YET_RECRUITING

Yangsan, Gyeongsangnam, South Korea
Rare Disease Research
RECRUITING

Atlanta, Georgia, 30329, United States
Rare Disease Research, LLC - FL
RECRUITING

Kissimmee, Florida, 34746, United States
S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital
NOT_YET_RECRUITING

Izmir, 11794, Turkey (Türkiye)
Seoul National University Hospital
NOT_YET_RECRUITING

Seoul, South Korea
Shiga General Hospital
NOT_YET_RECRUITING

Moriyama-shi, Shiga, 524-8524, Japan
Starship Children's Hospital
NOT_YET_RECRUITING

Auckland, 1023, New Zealand
The Children's Hospital of Philadelphia (CHOP)
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
UT Southwestern/Children's Health
RECRUITING

Dallas, Texas, 75207, United States
University of Kansas Medical Center (KUMC)
RECRUITING

Kansas City, Kansas, 66160, United States
University of Pittsburgh School of Medicine
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
Virginia Commonwealth University Health System
RECRUITING

Richmond, Virginia, 23298, United States
Yeditepe University Kosuyolu Hospital
NOT_YET_RECRUITING

Istanbul, 34718, Turkey (Türkiye)

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