Gene therapy may let severe hemophilia patients make their own clotting factor
NCT ID NCT03588299
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 25 times
Summary
This study tests a gene therapy called BAY2599023 in 11 adults with severe hemophilia A. The treatment delivers a healthy copy of the factor VIII gene so the body can produce its own clotting protein. The goal is to find the safest and most effective dose to reduce or stop bleeding episodes.
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.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
APHP-Hopital Necker Enfants malades
Paris, Île-de-France Region, 75015, France
-
Academisch Medisch Centrum (AMC)
Amsterdam, North Holland, 1105 AZ, Netherlands
-
Arkansas Children's Hospital - Hematology / Oncology
Little Rock, Arkansas, 72202, United States
-
C.S. Mott Children's Hospital - Hematology / Oncology
Ann Arbor, Michigan, 48109, United States
-
CHU Rennes - Hopital Pontchaillou
Rennes, Brittany Region, 35033, France
-
Erasmus Medisch Centrum
Rotterdam, South Holland, 3015 CE, Netherlands
-
Manchester Royal Infirmary
Manchester, Greater Manchester, M13 9WL, United Kingdom
-
SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
Sofia, Sofia City Province, 1756, Bulgaria
-
UW Health Carbone Cancer Center
Madison, Wisconsin, 53792, United States
-
Universitair Medisch Centrum Groningen
Groningen, 9713 GZ, Netherlands
-
University Medical Center Utrecht
Utrecht, 3584 CX, Netherlands
-
Universitätsklinikum des Saarlandes
Homburg, Saarland, 66421, Germany
-
Vivantes Klinikum im Friedrichshain
Berlin, 10249, Germany
Conditions
Explore the condition pages connected to this study.