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"

NCTID	NCT03588299 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Active
Indication	Hemophilia A
Disease Ontology Term	DOID:12134
Compound Name	DTX201
Compound Alias	Peboctocogene camaparvovec, BAY 2599023
Compound Description	liver-specific TTR promotor
Sponsor	Bayer
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	11
Results Posted	Not Available

Target Gene/Variant	F8
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVhu37
Editor Type	none
Dose 1	0.5E13 GC/kg
Dose 2	1E13 GC/kg
Dose 3	2E13 GC/kg
Dose 4	4E13 GC/kg
Dose 5

No.of Trial Sites	13
Locations	Netherlands,United States,United Kingdom,Bulgaria,France,Germany

Has US IND	True
FDA Designations
Recent Updates	License reverted to Ultragenyx in 2022, uncertain development status