Gene therapy breakthrough for hemophilia a patients
NCT ID NCT04323098
First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 30 times
Summary
This study tested a one-time gene therapy (valoctocogene roxaparvovec) combined with steroids in 22 men with severe hemophilia A. The goal was to raise their low clotting factor levels and reduce the need for regular factor infusions. Results showed increased factor activity and fewer bleeding episodes, offering a potential long-term management option.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for HEMOPHILIA A are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
Alfred Hospital
Melbourne, Australia
-
Campinas University Clinical Hospital
Campinas, Brazil
-
Changhua Christian Medical Foundation Changhua Christian Hospital
Changhua, Taiwan
-
Fiona Stanley Hospital
Perth, Australia
-
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo, Brazil
-
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
-
Royal Brisbane and Women's Hospital
Brisbane, Australia
-
Royal Prince Alfred Hospital
Sydney, Australia
-
Taichung Veterans General Hospital
Taichung, Taiwan
-
The Royal Adelaide Hospital
Adelaide, Australia
-
University of California Davis Health
Sacramento, California, 95817, United States
-
Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.