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NS-050/NCNP-03 in Boys With DMD (Meteor50)
Sponsor: NS Pharma, Inc.
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.
Official title: 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)
Key Details
Gender
MALE
Age Range
4 Years - 14 Years
Study Type
INTERVENTIONAL
Enrollment
20
Start Date
2024-09-18
Completion Date
2027-05
Last Updated
2025-09-10
Healthy Volunteers
No
Conditions
Interventions
NS-050/NCNP-03
NS-050/NCNP-03 solution for IV infusion.
Placebo
NS-050/NCNP-03 placebo-matching solution for IV infusion.
Locations (18)
Children's Hospital Los Angeles
Los Angeles, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Alberta Children's Hospital
Calgary, Alberta, Canada
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
London Health Sciences Centre
London, Ontario, Canada
National Hospital Organization Nagara Medical Center
Nagara, Gifu, Japan
Hyogo Medical University Hospital
Nishinomiya, Hyōgo, Japan
NHO Osaka Toneyama Medical Center
Toyonaka, Osaka, Japan
National Center of Neurology and Psychiatry
Kodaira, Tokyo, Japan
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, South Korea
Seoul National University Hospital
Seoul, South Korea
Ankara Bilkent City Hospital
Ankara, Turkey (Türkiye)
Istanbul University
Istanbul, Turkey (Türkiye)
Yeditepe University Kosuyolu Hospital
Istanbul, Turkey (Türkiye)