New hope for boys with DMD: weekly infusion aims to restore muscle protein
NCT ID NCT05996003
Summary
This study is testing a new drug called NS-089/NCNP-02 for boys with Duchenne muscular dystrophy (DMD) caused by a specific genetic error (exon 44). The drug is given as a weekly IV infusion and aims to help muscles produce more of a crucial protein called dystrophin. Researchers will measure if the drug is safe, how the body processes it, and if it improves muscle function and strength in 20 boys aged 4 to 15 who can still walk.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Alberta Children's Hospital
NOT_YET_RECRUITINGCalgary, Alberta, Canada
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Ankara Bilkent City Hospital
NOT_YET_RECRUITINGAnkara, 06800, Turkey (Türkiye)
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Ann and Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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British Columbia Children's Hospital
NOT_YET_RECRUITINGVancouver, British Columbia, Canada
Contact Email: •••••@•••••
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
Contact Email: •••••@•••••
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Fukui Prefectural Hospital
NOT_YET_RECRUITINGFukui-shi, Fukui, 910-8526, Japan
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London Health Sciences Centre
RECRUITINGLondon, Ontario, Canada
Contact Email: •••••@•••••
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NHO Osaka Toneyama Medical Center
NOT_YET_RECRUITINGToyonaka, Osaka, 560-8552, Japan
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National Center of Neurology and Psychiatry
NOT_YET_RECRUITINGKodaira, Tokyo, 187-8551, Japan
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National Hospital Organization Nagara Medical Center
RECRUITINGNagara, Gifu-shi, Gifu, 502-8558, Japan
Contact Email: •••••@•••••
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Perth Children's Hospital
NOT_YET_RECRUITINGNedlands, Western Australia, 6009, Australia
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Pusan National University Yangsan Hospital
NOT_YET_RECRUITINGYangsan, Gyeongsangnam, South Korea
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 30329, United States
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Rare Disease Research, LLC - FL
RECRUITINGKissimmee, Florida, 34746, United States
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S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital
NOT_YET_RECRUITINGIzmir, 11794, Turkey (Türkiye)
Contact Email: •••••@•••••
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Seoul National University Hospital
NOT_YET_RECRUITINGSeoul, South Korea
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Shiga General Hospital
NOT_YET_RECRUITINGMoriyama-shi, Shiga, 524-8524, Japan
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Starship Children's Hospital
NOT_YET_RECRUITINGAuckland, 1023, New Zealand
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The Children's Hospital of Philadelphia (CHOP)
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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UT Southwestern/Children's Health
RECRUITINGDallas, Texas, 75207, United States
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University of Kansas Medical Center (KUMC)
RECRUITINGKansas City, Kansas, 66160, United States
Contact Email: •••••@•••••
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University of Pittsburgh School of Medicine
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
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Virginia Commonwealth University Health System
RECRUITINGRichmond, Virginia, 23298, United States
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Yeditepe University Kosuyolu Hospital
NOT_YET_RECRUITINGIstanbul, 34718, Turkey (Türkiye)
Contact Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.