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

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Updated on 16 June 2022

See if I qualify

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 Identifier	NCT03588299
Sponsor	Bayer
Last Modified on	16 June 2022

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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

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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".