Last updated on September 2020

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201) a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered Non-infectious Virus (AAV) as a "Shuttle".


Brief description of study

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.

Clinical Study Identifier: NCT03588299

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Arkansas Children's Hospital

Little Rock, AR United States
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Loma Linda Children's Hospital

San Bernardino, CA United States
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C.S. Mott Children's Hospital

Ann Arbor, MI United States
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St Thomas' Hospital

London, United Kingdom
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Manchester Royal Infirmary

Manchester, United Kingdom
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Recruitment Status: Open


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