Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
20 participants
INTERVENTIONAL
2024-09-18
2027-05-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Part 1: NS-050/NCNP-03
Participants will be randomized and receive NS-050/NCNP-03 intravenous (IV) infusions once weekly for 2 weeks at each of MAD levels (1.95, 5, 10, 20, 40, and 80 mg/kg).
NS-050/NCNP-03
NS-050/NCNP-03 solution for IV infusion.
Part 1: Placebo
Participants will be randomized and receive NS-050/NCNP-03 placebo-matching IV infusions once weekly for 2 weeks at each of MAD levels.
Placebo
NS-050/NCNP-03 placebo-matching solution for IV infusion.
Part 2: NS-050/NCNP-03
Participants will receive NS-050/NCNP-03 IV infusions once weekly for 24 weeks at the dosage selected by the Data and Safety Monitoring Board (DSMB) at the conclusion of Part 1.
NS-050/NCNP-03
NS-050/NCNP-03 solution for IV infusion.
Interventions
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NS-050/NCNP-03
NS-050/NCNP-03 solution for IV infusion.
Placebo
NS-050/NCNP-03 placebo-matching solution for IV infusion.
Eligibility Criteria
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Inclusion Criteria
* 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 Criteria
* 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.
4 Years
14 Years
MALE
No
Sponsors
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Nippon Shinyaku Co., Ltd.
INDUSTRY
NS Pharma, Inc.
INDUSTRY
Responsible Party
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Locations
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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)
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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NS-050/NCNP-03-101
Identifier Type: -
Identifier Source: org_study_id
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