One-Time gene infusion could free patients from lifelong bleeding disorder treatments
NCT ID NCT06224907
Summary
This study is testing a single-dose gene therapy called valoctocogene roxaparvovec (BMN 270) for Japanese adults with severe hemophilia A. The goal is to see if this one-time intravenous treatment can help the body produce its own blood-clotting factor, reducing or eliminating the need for regular, lifelong infusions of replacement factor. Researchers will measure changes in clotting factor levels, bleeding rates, and quality of life over a year.
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Contacts and locations
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Locations
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Asahikawa Medical University Hospital
Hokkaido, Asahikawa, 078-8510, Japan
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Nagoya University Hospital
Aichi, Nagoya, 466-8560, Japan
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Saitama Medical University Hospital
Saitama, Iruma-gun, 350-0495, Japan
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Tokyo Medical University Hospital
Tokyo, Shinjuku-ku, 160-0023, Japan
Conditions
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