Brief Title
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".
Official Title
A Phase 1/2 Open-label Safety and Dose-finding Study of BAY2599023 (DTX201), an Adeno-associated Virus (AAV) hu37-mediated Gene Transfer of B-domain Deleted Human Factor VIII, in Adults With Severe Hemophilia A
Brief 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.
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interest
Secondary Outcome
Expression pattern of FVIII activity.
Condition
Hemophilia A
Intervention
BAY2599023 (DTX201)
Study Arms / Comparison Groups
BAY2599023 / (DTX201)
Description: Adult patients with severe hemophilia A, who have been previously treated with FVIII products
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
11
Start Date
November 7, 2018
Completion Date
November 30, 2026
Primary Completion Date
November 3, 2026
Eligibility 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
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
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Location Countries
Bulgaria
Location Countries
Bulgaria
Administrative Informations
NCT ID
NCT03588299
Organization ID
19429
Secondary IDs
2017-000806-39
Responsible Party
Sponsor
Study Sponsor
Bayer
Collaborators
Ultragenix pharmaceutical
Study Sponsor
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Verification Date
September 2022