NS-050/NCNP-03 in Boys With DMD (Meteor50)

Running, not enrolling · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy

In brief

This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.

Key facts

Study ID

NCT06053814

Run by

NS Pharma, Inc.

People needed

20

Starts

2024-09-18

Expected to finish

2028-03-01

Last updated by the study team

2026-06-16

Who can join

Age: 4 and older, up to 15. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Children's Hospital Los Angeles — Los Angeles, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States
• Alberta Children's Hospital — Calgary, Alberta, Canada
• British Columbia Children's Hospital — Vancouver, British Columbia, Canada
• London Health Sciences Centre — London, Ontario, Canada
• National Hospital Organization Nagara Medical Center — Nagara, Gifu, Japan
• Hyogo Medical University Hospital — Nishinomiya, Hyōgo, Japan
• Miyagi Children's Hospital — Sendai, Miyagi, Japan
• NHO Osaka Toneyama Medical Center — Toyonaka, Osaka, Japan
• National Center of Neurology and Psychiatry — Kodaira, Tokyo, Japan
• Pusan National University Yangsan Hospital — Yangsan, Gyeongsangnam-do, South Korea
• Seoul National University Hospital — Seoul, South Korea
• Ankara Bilkent City Hospital — Ankara, Turkey (Türkiye)
• Istanbul University — Istanbul, Turkey (Türkiye)
• Yeditepe University Kosuyolu Hospital — Istanbul, Turkey (Türkiye)

Full record on ClinicalTrials.gov

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