One-Shot gene therapy aims to free patients from lifelong injections
NCT ID NCT06224907
Summary
This study is testing a single-dose gene therapy called BMN 270 for Japanese adults with severe Hemophilia A. The goal is to see if the therapy can help the body produce its own missing clotting factor, potentially reducing or eliminating the need for regular preventative injections. Researchers will measure changes in 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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