Status:
RECRUITING
NS-050/NCNP-03 in Boys With DMD (Meteor50)
Lead Sponsor:
NS Pharma, Inc.
Collaborating Sponsors:
Nippon Shinyaku Co., Ltd.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
4-14 years
Phase:
PHASE1
PHASE2
Brief 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...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
September 18 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2027
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT06053814
Start Date
September 18 2024
End Date
May 1 2027
Last Update
September 10 2025
Active Locations (18)
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1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80011
3
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
4
University of Kansas Medical Center
Kansas City, Kansas, United States, 66103