Treatment for boys with Duchenne Muscular Dystrophy using NS-050/NCNP-03

A Phase 1/2, First in Human, Multiple-dose, 2-part Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-050/NCNP-03 in Boys With Duchenne Muscular Dystrophy (DMD)

This study is testing a new treatment called NS-050/NCNP-03 to see if it can help boys with Duchenne Muscular Dystrophy who have a specific gene change.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 4 Years to 14 Years
• Sex: Male
• Sponsor: NS Pharma, Inc. (industry)
• Locations: 11 sites (Los Angeles, California and 10 other locations)
• Trial ID: NCT06053814 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety and efficacy of NS-050/NCNP-03 in boys with Duchenne Muscular Dystrophy (DMD) who have a specific exon deletion. It involves a Phase 1/2 design with multiple ascending doses administered weekly for 12 weeks, followed by an open-label treatment phase lasting 24 weeks. Participants must be able to walk independently and meet specific eligibility criteria related to their condition and treatment history.

Who should consider this trial

Good fit: Ideal candidates are male patients aged 4 to 15 years with a confirmed DMD exon deletion amenable to exon 50 skipping.

Not a fit: Patients with symptomatic cardiomyopathy or those currently on other investigational drugs may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve muscle function and quality of life for boys with DMD.

How similar studies have performed: Other studies targeting exon skipping in DMD have shown promise, indicating potential for success with this approach.

Eligibility criteria

Inclusion Criteria:

* Male ≥ 4 years and \<15 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.

Where this trial is running

Los Angeles, California and 10 other locations

• Children's Hospital Los Angeles — Los Angeles, California, United States (NOT_YET_RECRUITING)
• Children's Hospital Colorado — Aurora, Colorado, United States (RECRUITING)
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (RECRUITING)
• University of Kansas Medical Center — Kansas City, Kansas, United States (RECRUITING)
• Shriners Children's Portland — Portland, Oregon, United States (NOT_YET_RECRUITING)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (RECRUITING)
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States (NOT_YET_RECRUITING)
• National Hospital Organization Nagara Medical Center — Nagara, Gifu, Japan (RECRUITING)
• Hyogo Medical University Hospital — Nishinomiya, Hyogo, Japan (RECRUITING)
• NHO Osaka Toneyama Medical Center — Toyonaka, Osaka, Japan (RECRUITING)
• National Center of Neurology and Psychiatry — Kodaira, Tokyo, Japan (RECRUITING)

Study contacts

• Study coordinator: Trial Info

Conditions: Duchenne Muscular Dystrophy