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NCT03588299 · ClinicalTrials.gov record · last posted 2026-07-06

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"

StatusActive, Not Recruiting
PhasePhase 1/2
SponsorBayer
Started2018-11-07
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from the ClinicalTrials.gov record
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.

Protocol amendment history 2 changes detected by DataLookout

2026-06-16
minor
Primary completion moved earlier: 2026-11-03 → 2026-09-15
2026-06-16
minor
Completion moved earlier: 2026-11-03 → 2026-09-15
Trial Details
NCT Number NCT03588299
Lead Sponsor Bayer
Collaborators: Ultragenix pharmaceutical
Conditions Hemophilia A
Enrollment 11 participants
Start Date 2018-11-07
Primary Completion 2026-09-15 (estimated)
Study Completion 2026-09-15 (estimated)
Updated on ClinicalTrials.gov 2026-07-06