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".

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: Hemophilia | Hemophilia A
  • Age: Between 18 - 100 Years
  • Gender: Male

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.
  • Current evidence of measurable inhibitor against factor VIII, as assessed by the central laboratory and/or prior history of inhibitors to FVIII protein.
  • 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/mm3; HIV+ and stable participants with CD4 count >200/mm3 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.

Recruitment Status: Open

Brief Description Eligibility Contact Research Team

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