One-time gene therapy may free hemophilia patients from regular infusions
NCT ID NCT06224907
First seen Mar 26, 2026 · Last updated Apr 23, 2026 · Updated 3 times
Summary
This study tests a one-time gene therapy (valoctocogene roxaparvovec) in 6 Japanese adults with severe hemophilia A. The goal is to see if it can raise clotting factor levels and reduce or eliminate the need for regular factor VIII infusions. Participants must be on preventive factor therapy before joining. The study measures safety and how well the treatment works over about a year.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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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