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New hope for duchenne: experimental drug aims to restore muscle protein

NCT ID NCT06053814

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial is testing a drug called NS-050/NCNP-03 in 20 boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The drug is designed to skip a faulty section of the dystrophin gene, allowing the body to produce a shorter but still functional version of the dystrophin protein, which is essential for muscle strength. The study will first assess safety and dosing, then measure changes in dystrophin levels and muscle function over several months.

What this could mean

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

Active substance
NS-050/NCNP-03 (a drug designed to skip exon 50 of the dystrophin gene, helping produce a shorter but functional dystrophin protein)
What this could lead to
If successful, this drug could help boys with a specific DMD mutation produce a working version of dystrophin, potentially slowing muscle decline and improving quality of life.
What could go wrong
This is an early phase 1/2 trial with only 20 participants, so safety and effectiveness are not yet proven. The drug may not produce enough dystrophin to make a meaningful difference, and side effects are unknown.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 20 people

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

Started

Sep 2024

Expected to finish

Mar 2028

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 15 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 \<16 years of age; * Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame; * Able to walk independently without assistive devices; * Able 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: * Evidence of symptomatic cardiomyopathy; * Current or previous treatment with anabolic steroids (e.g., oxendolone, oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug; * Currently taking another investigational drug or has taken another investigational drug within 3 months prior to the first dose of study drug; * Surgery within the 3 months prior to the first dose of study drug or planned during the study duration; * Having taken any gene therapy. 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.

Duchenne muscular dystrophy

Contacts and locations

Locations

  • Alberta Children's Hospital

    Calgary, Alberta, Canada

  • Ankara Bilkent City Hospital

    Ankara, Turkey (Türkiye)

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

    Chicago, Illinois, 60611, United States

  • British Columbia Children's Hospital

    Vancouver, British Columbia, Canada

  • Children's Hospital Colorado

    Aurora, Colorado, 80011, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Hyogo Medical University Hospital

    Nishinomiya, Hyōgo, 663-8501, Japan

  • Istanbul University

    Istanbul, Turkey (Türkiye)

  • London Health Sciences Centre

    London, Ontario, Canada

  • Miyagi Children's Hospital

    Sendai, Miyagi, 989-3126, Japan

  • 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, 502-8558, Japan

  • Pusan National University Yangsan Hospital

    Yangsan, Gyeongsangnam-do, South Korea

  • Seoul National University Hospital

    Seoul, South Korea

  • University of Kansas Medical Center

    Kansas City, Kansas, 66103, United States

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213, United States

  • Yeditepe University Kosuyolu Hospital

    Istanbul, Turkey (Türkiye)

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