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

Prior treatment

• 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.

Other

• Evidence of symptomatic cardiomyopathy;

• 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.