NS-050/NCNP-03 in Boys With DMD (Meteor50)
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)
NS Pharma, Inc.
20 participants
Sep 18, 2024
INTERVENTIONAL
Conditions
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 exon deletion amenable to exon 50 skipping.
Eligibility
Inclusion Criteria5
- 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.
Exclusion Criteria5
- 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.
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Interventions
NS-050/NCNP-03 solution for IV infusion.
NS-050/NCNP-03 placebo-matching solution for IV infusion.
Locations(18)
View Full Details on ClinicalTrials.gov
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NCT06053814