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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03588299
Other study ID # 19429
Secondary ID 2023-505827-29-0
Status Active, not recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date November 7, 2018
Est. completion date November 30, 2026

Study information

Verified date June 2024
Source Bayer
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 11
Est. completion date November 30, 2026
Est. primary completion date November 3, 2026
Accepts healthy volunteers No
Gender Male
Age group 18 Years and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
BAY2599023 (DTX201)
Single escalating doses with 4 dose steps; Single intravenous (IV) administration.

Locations

Country Name City State
Bulgaria SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD Sofia
France Hopital Necker les enfants malades - Paris Paris
France Hôpital Pontchaillou Rennes Cedex
Germany Vivantes Klinikum im Friedrichshain Berlin
Germany Universitätsklinikum des Saarlandes Homburg Saarland
Netherlands Academisch Medisch Centrum (AMC) Amsterdam
Netherlands Universitair Medisch Centrum Groningen Groningen
Netherlands Erasmus Medisch Centrum Rotterdam
Netherlands University Medical Center Utrecht Utrecht
United Kingdom Manchester Royal Infirmary Manchester
United States C.S. Mott Children's Hospital Ann Arbor Michigan
United States Arkansas Children's Hospital Little Rock Arkansas
United States University of Wisconsin - Madison Madison Wisconsin

Sponsors (2)

Lead Sponsor Collaborator
Bayer Ultragenix pharmaceutical

Countries where clinical trial is conducted

United States,  Bulgaria,  France,  Germany,  Netherlands,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interest Up to 5 years
Secondary Expression pattern of FVIII activity. Determined using both a one-stage assay and chromogenic assay. Up to 5 years
Secondary Proportion of patients in the respective dose step, that reached an expression of FVIII above 5% At 6 months and 12 months following the IV administration of BAY2599023
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