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"

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Hemophilia A

In brief

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.

Key facts

Study ID: NCT03588299
Run by: Bayer
People needed: 11
Starts: 2018-11-07
Expected to finish: 2026-09-15
Last updated by the study team: 2026-07-06

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

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

Where it is running

Arkansas Children's Hospital - Hematology / Oncology — Little Rock, Arkansas, United States
C.S. Mott Children's Hospital - Hematology / Oncology — Ann Arbor, Michigan, United States
UW Health Carbone Cancer Center — Madison, Wisconsin, United States
SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD — Sofia, Sofia City Province, Bulgaria
CHU Rennes - Hopital Pontchaillou — Rennes, Brittany Region, France
APHP-Hopital Necker Enfants malades — Paris, Île-de-France Region, France
Universitätsklinikum des Saarlandes — Homburg, Saarland, Germany
Vivantes Klinikum im Friedrichshain — Berlin, Germany
Academisch Medisch Centrum (AMC) — Amsterdam, North Holland, Netherlands
Erasmus Medisch Centrum — Rotterdam, South Holland, Netherlands
Universitair Medisch Centrum Groningen — Groningen, Netherlands
University Medical Center Utrecht — Utrecht, Netherlands
Manchester Royal Infirmary — Manchester, Greater Manchester, United Kingdom

Full record on ClinicalTrials.gov

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