Study Summary
In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the disease. By replacing the defective gene with a healthy copy the human body may produce clotting factor on its own. Hemophilia A is a bleeding disorder in which the human body does not have enough clotting factor VIII, a protein that controls bleeding. Researcher want to find the optimal dose of BAY 2599023 (DTX201) so that the body may produce enough clotting factor on its own.
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Request More InfoInterventions
BAY2599023 (DTX201)DRUG
Single escalating doses with 4 dose steps; Single intravenous (IV) administration.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Arkansas Children's Hospital - Hematology / Oncology | Little Rock | Arkansas | United States |
| C.S. Mott Children's Hospital - Hematology / Oncology | Ann Arbor | Michigan | United States |
| UW Health Carbone Cancer Center | Madison | Wisconsin | United States |
| SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD | Sofia | Sofia City Province | Bulgaria |
| CHU Rennes - Hopital Pontchaillou | Rennes | Brittany Region | France |
| APHP-Hopital Necker Enfants malades | Paris | Île-de-France Region | France |
| Universitätsklinikum des Saarlandes | Homburg | Saarland | Germany |
| Vivantes Klinikum im Friedrichshain | Berlin | Germany | |
| Academisch Medisch Centrum (AMC) | Amsterdam | North Holland | Netherlands |
| Erasmus Medisch Centrum | Rotterdam | South Holland | Netherlands |
| Universitair Medisch Centrum Groningen | Groningen | Netherlands | |
| University Medical Center Utrecht | Utrecht | Netherlands | |
| Manchester Royal Infirmary | Manchester | Greater Manchester | United Kingdom |