do_not_disturb_altRecruitment Complete
Hemophilia A
Bayer Identifier:
19429
ClinicalTrials.gov Identifier:
EudraCT Number:
EU CT Number:
2023-505827-29-00
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"
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.
Key Participants Requirements
Sex
MaleAge
18 - N/ATrial summary
Enrollment Goal
11Trial Dates
November 2018 - November 2026Phase
Phase 1/Phase 2Could I Receive a placebo
NoProducts
BAY2599023 (DTX201)Accepts Healthy Volunteer
NoWhere to participate
Status | Institution | Location |
---|---|---|
Completed | C.S. Mott Children's Hospital - Hematology / Oncology | Ann Arbor, 48109, United States |
Completed | SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD | Sofia, 1756, Bulgaria |
Completed | Arkansas Children's Hospital - Hematology / Oncology | Little Rock, 72202, United States |
Withdrawn | Universitätsklinikum der Johann Wolfgang Goethe Universität | Frankfurt, 60590, Germany |
Active, not recruiting | Universitätsklinikum des Saarlandes | Homburg, 66421, Germany |
Completed | Vivantes Klinikum im Friedrichshain | Berlin, 10249, Germany |
Completed | Manchester Royal Infirmary | Manchester, M13 9WL, United Kingdom |
Withdrawn | St Thomas' Hospital | London, SE1 7EH, United Kingdom |
Active, not recruiting | Academisch Medisch Centrum (AMC) | AMSTERDAM, 1105 AZ, Netherlands |
Completed | Erasmus Medisch Centrum | ROTTERDAM, 3015 CE, Netherlands |
Completed | Universitair Medisch Centrum Groningen | GRONINGEN, 9713 GZ, Netherlands |
Completed | University Medical Center Utrecht | UTRECHT, 3584 CX, Netherlands |
Completed | Hôpital Pontchaillou | RENNES CEDEX, 35033, France |
Withdrawn | Hôpital de la Timone - Marseille | MARSEILLE, 13005, France |
Active, not recruiting | Hopital Necker les enfants malades - Paris | PARIS, 75015, France |
Withdrawn | Hôpital Calmette - Lille Cedex | LILLE CEDEX, 59037, France |
Withdrawn | Hospital Regional de Málaga | Málaga, 29010, Spain |
Withdrawn | Hospital Central de Asturias | Oviedo, 33011, Spain |
Withdrawn | Hospital de la Santa Creu i Sant Pau | Gynecology Department | Barcelona, 08041, Spain |
Withdrawn | Ciutat Sanitaria i Universitaria de la Vall d'Hebron | Barcelona, 08035, Spain |
Withdrawn | Hospital Universitario La Paz | IdiPAZ - Neurology Department | Madrid, 28046, Spain |
Withdrawn | Hospital Universitari i Politècnic La Fe | Valencia, 46026, Spain |
Withdrawn | University Hospitals Cleveland Medical Center | Cleveland, 44106, United States |
Active, not recruiting | University of Wisconsin - Madison | Madison, 53792, United States |
Withdrawn | Loma Linda Children's Hospital | San Bernardino, 92408, United States |
Primary Outcome
- Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interestdate_rangeTime Frame:Up to 5 years
Secondary Outcome
- Expression pattern of FVIII activity.Determined using both a one-stage assay and chromogenic assay.date_rangeTime Frame:Up to 5 years
- Proportion of patients in the respective dose step, that reached an expression of FVIII above 5%date_rangeTime Frame:At 6 months and 12 months following the IV administration of BAY2599023
Trial design
Trial Type
InterventionalIntervention Type
DrugTrial Purpose
TreatmentAllocation
N/ABlinding
N/AAssignment
Single Group AssignmentTrial Arms
1