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".

  • STATUS
    Recruiting
  • End date
    Jun 28, 2027
  • participants needed
    30
  • sponsor
    Bayer
Updated on 13 May 2022
coagulation factor
bleeding tendency
severe haemophilia a
antihemophilic factor

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.

Details
Condition Hemophilia A
Treatment BAY2599023 (DTX201)
Clinical Study IdentifierNCT03588299
SponsorBayer
Last Modified on13 May 2022

Eligibility

Yes No Not Sure

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/m2
Evidence of active hepatitis B or C
Current evidence of measurable inhibitor against factor VIII, prior history of
Currently on antiviral therapy for hepatitis B or C
inhibitors to FVIII protein or clinical history suggestive of inhibitor
Significant underlying liver disease
Detectable antibodies reactive with AAVhu37capsid
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
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
Clear my responses

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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