Phase
Condition
Muscular Dystrophy
Treatment
NS-050/NCNP-03
Placebo
Clinical Study ID
Ages 4-14 Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Male ≥ 4 years and <15 years of age;
Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping ofexon 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 toremain on a stable dose for the duration of the study.
Other inclusion criteria may apply.
Exclusion
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 priorto first dose of study drug;
Currently taking another investigational drug or has taken another investigationaldrug 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 duringthe study duration;
Having taken any gene therapy.
Other exclusion criteria may apply.
Study Design
Connect with a study center
National Hospital Organization Nagara Medical Center
Nagara, Gifu 502-8558
JapanActive - Recruiting
Hyogo Medical University Hospital
Nishinomiya, Hyogo 663-8501
JapanActive - Recruiting
NHO Osaka Toneyama Medical Center
Toyonaka, Osaka 560-8552
JapanActive - Recruiting
National Center of Neurology and Psychiatry
Kodaira, Tokyo 187-8551
JapanActive - Recruiting
Children's Hospital Los Angeles
Los Angeles, California 90027
United StatesSite Not Available
Children's Hospital Colorado
Aurora, Colorado 80011
United StatesActive - Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United StatesActive - Recruiting
University of Kansas Medical Center
Kansas City, Kansas 66103
United StatesActive - Recruiting
Shriners Children's Portland
Portland, Oregon 97239
United StatesSite Not Available
Shriners Hospital for Children
Portland, Oregon 97239
United StatesSite Not Available
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesActive - Recruiting
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania 15213
United StatesSite Not Available
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