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

• Status: Not yet recruiting
• Trial end date: 2027-05-01
• Target enrollment: 0
• Participant gender: Male

Summary

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 mutation amenable to exon 50 skipping.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: NS Pharma, Inc.
• Collaborator: Nippon Shinyaku Co., Ltd.

Criteria

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 50 to restore the dystrophin mRNA reading frame;

• Able to walk independently without assistive devices;

• Able to complete the TTSTAND without assistance in <7 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.