Trial Condition(s):
Hemophilia A
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".
Bayer Identifier:
19429
ClinicalTrials.gov Identifier:
EudraCT Number:
EU CT Number:
2023-505827-29-00
Trial Purpose
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.
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
Trial Summary
Enrollment Goal
Trial Dates
Phase
Could I receive a placebo?
Products
Accepts Healthy Volunteers
Where to Participate
| Locations | |
|---|---|
Locations C.S. Mott Children's Hospital Ann Arbor, United States, 48109 | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD Sofia, Bulgaria, 1756 | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations Arkansas Children's Hospital Little Rock, United States, 72202-3500 | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations Universitätsklinikum des Saarlandes Homburg, Germany, 66421 | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations Vivantes Klinikum im Friedrichshain Berlin, Germany, 10249 | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations Manchester Royal Infirmary Manchester, United Kingdom, M13 9WL | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations Academisch Medisch Centrum (AMC) AMSTERDAM, Netherlands, 1105 AZ | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations Erasmus Medisch Centrum ROTTERDAM, Netherlands, 3015 CE | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations Universitair Medisch Centrum Groningen GRONINGEN, Netherlands, 9713 GZ | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations University Medical Center Utrecht UTRECHT, Netherlands, 3584 CX | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations Hôpital Pontchaillou RENNES CEDEX, France, 35033 | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations Hopital Necker les enfants malades - Paris PARIS, France, 75015 | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Locations University of Wisconsin - Madison Madison, United States, 53792 | Contact Us: E-mail: [email protected] Phone: (+) 1-888-8422937 |
Trial Design
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
Trial Type:
Interventional
Intervention Type:
Drug
Trial Purpose:
Treatment
Allocation:
N/A
Blinding:
N/A
Assignment:
Single Group Assignment
Trial Arms:
1
Primary Outcome
- Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interestTimeframe: Up to 5 years
Secondary Outcome
- Expression pattern of FVIII activity.Determined using both a one-stage assay and chromogenic assay.Timeframe: Up to 5 years
- Proportion of patients in the respective dose step, that reached an expression of FVIII above 5%Timeframe: At 6 months and 12 months following the IV administration of BAY2599023
Additional Information
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