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 Altere

• Status: Recruiting
• Trial end date: 2027-06-28
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bayer
• Collaborators: Ultragenix pharmaceutical; Ultragenyx Pharmaceutical Inc
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

• Males age 18 years or older.

• Confirmed diagnosis of hemophilia A as evidenced by their medical history with plasma FVIII activity levels < 1% of normal or at screening.

• Have >150 exposure days (EDs) to FVIII concentrates (recombinant or plasma-derived).

If on prophylaxis, are required to be willing to stop prophylactic treatment at specified time points throughout the study or If on-demand: should have had > 4 bleeding events in the last 52 weeks

• Agree to use reliable barrier contraception.

Exclusion Criteria:

• History of allergic reaction to any FVIII product.

• Clinically relevant findings in the physical examination considered critical by the treating physician, including obesity with BMI > 35 kg/m*2

• Current evidence of measurable inhibitor against factor VIII, prior history of inhibitors to FVIII protein or clinical history suggestive of inhibitor.

• Evidence of active hepatitis B or C.

• Currently on antiviral therapy for hepatitis B or C.

• Significant underlying liver disease.

• Serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm*3; HIV+ and stable participants with CD4 count >200/mm*3 and undetectable viral load are eligible to enroll.

• Detectable antibodies reactive with AAVhu37capsid.

• Participant with another bleeding disorder that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B).

• Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational product within the last 12 weeks.

• Known or suspected hypersensitivity or allergic reaction to trial product(s) or related FVIII products or any component of BAY2599023 (DTX201), or a contraindication to prednisolone