NCT03588299

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"

A Phase 1/2 Open-label Safety and Dose-finding Study of BAY2599023 (DTX201), an Adeno-associated Virus (AAV) hu37-mediated Gene Transfer of B-domain Deleted Human Factor VIII, in Adults With Severe Hemophilia A

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Bayer · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	BAY2599023 (DTX201)	Single escalating doses with 4 dose steps; Single intravenous (IV) administration.

Timeline

• Start date: 2018-11-07
• Primary completion: 2026-11-03
• Completion: 2026-11-03
• First posted: 2018-07-17
• Last updated: 2026-04-17

Locations

13 sites across 6 countries: United States, Bulgaria, France, Germany, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study