One-Time gene therapy could free hemophilia patients from frequent infusions
NCT ID NCT04323098
First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 28 times
Summary
This study tested a one-time gene therapy (valoctocogene roxaparvovec) in 22 adults with severe hemophilia A. The goal was to help their bodies produce their own clotting factor VIII, reducing or stopping the need for regular factor infusions. Participants also received steroids to manage immune reactions. Results showed increased factor VIII levels and less bleeding, but long-term effects and need for ongoing monitoring remain.
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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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Alfred Hospital
Melbourne, Australia
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Campinas University Clinical Hospital
Campinas, Brazil
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Changhua Christian Medical Foundation Changhua Christian Hospital
Changhua, Taiwan
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Fiona Stanley Hospital
Perth, Australia
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Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo, Brazil
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Royal Brisbane and Women's Hospital
Brisbane, Australia
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Royal Prince Alfred Hospital
Sydney, Australia
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Taichung Veterans General Hospital
Taichung, Taiwan
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The Royal Adelaide Hospital
Adelaide, Australia
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University of California Davis Health
Sacramento, California, 95817, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
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