One-Time gene therapy shows promise for hemophilia a
NCT ID NCT04323098
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a one-time gene therapy (valoctocogene roxaparvovec) in 22 adults with severe hemophilia A. The treatment aims to help the body produce its own clotting factor VIII, reducing the need for regular infusions. Participants also received steroids to manage immune reactions. Results showed increased factor VIII levels and less need for replacement therapy.
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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