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New hope for duchenne: experimental drug targets genetic glitch

NCT ID NCT05996003

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Sep 16, 2026 · Updated 3 times

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 20 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2024

Expected to finish

Feb 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 to 14 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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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

    Calgary, Alberta, Canada

  • Ankara Bilkent City Hospital

    Ankara, 06800, Turkey (Türkiye)

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

    Chicago, Illinois, 60611, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • British Columbia Children's Hospital

    Vancouver, British Columbia, Canada

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Columbia University Pediatric Neuromuscular Center

    New York, New York, 10032, United States

  • Fukui Prefectural Hospital

    Fukui-shi, Fukui, 910-8526, Japan

  • Istanbul University- Istanbul Faculty of Medicine

    Istanbul, 34093, Turkey (Türkiye)

  • London Health Sciences Centre

    London, Ontario, Canada

  • NHO Osaka Toneyama Medical Center

    Toyonaka, Osaka, 560-8552, Japan

  • National Center of Neurology and Psychiatry

    Kodaira, Tokyo, 187-8551, Japan

  • National Hospital Organization Nagara Medical Center

    Nagara, Gifu-shi, Gifu, 502-8558, Japan

  • Perth Children's Hospital

    Nedlands, Western Australia, 6009, Australia

  • Pusan National University Yangsan Hospital

    Yangsan, Gyeongsangnam, South Korea

  • Queensland Children's Hospital

    South Brisbane, Queensland, 4101, Australia

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

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

    Izmir, 11794, Turkey (Türkiye)

  • Seoul National University Bundang Hospital

    Seongnam-si, Gyeonggi-do, 13620, South Korea

  • Seoul National University Hospital

    Seoul, South Korea

  • Shiga General Hospital

    Moriyama-shi, Shiga, 524-8524, Japan

  • Starship Children's Hospital

    Auckland, 1023, New Zealand

  • The Children's Hospital of Philadelphia (CHOP)

    Philadelphia, Pennsylvania, 19104, United States

  • UT Southwestern/Children's Health

    Dallas, Texas, 75207, United States

  • University of Kansas Medical Center (KUMC)

    Kansas City, Kansas, 66160, United States

  • University of Pittsburgh School of Medicine

    Pittsburgh, Pennsylvania, 15224, United States

  • Virginia Commonwealth University Health System

    Richmond, Virginia, 23298, United States

  • Yeditepe University Kosuyolu Hospital

    Istanbul, 34718, Turkey (Türkiye)

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Other studies related to the condition(s) this trial covers.