do_not_disturb_altRecruitment Complete

Hemophilia A

Bayer Identifier:

19429

ClinicalTrials.gov Identifier:

NCT03588299

EudraCT Number:

2017-000806-39

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

Male

Age

18 - N/A
  • - 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.
  • - 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
11
Trial Dates
November 2018 - November 2026
Phase
Phase 1/Phase 2
Could I Receive a placebo
No
Products
BAY2599023 (DTX201)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Completed
C.S. Mott Children's Hospital - Hematology / OncologyAnn Arbor, 48109, United States
Completed
SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EADSofia, 1756, Bulgaria
Completed
Arkansas Children's Hospital - Hematology / OncologyLittle Rock, 72202, United States
Withdrawn
Universitätsklinikum der Johann Wolfgang Goethe UniversitätFrankfurt, 60590, Germany
Active, not recruiting
Universitätsklinikum des SaarlandesHomburg, 66421, Germany
Completed
Vivantes Klinikum im FriedrichshainBerlin, 10249, Germany
Completed
Manchester Royal InfirmaryManchester, M13 9WL, United Kingdom
Withdrawn
St Thomas' HospitalLondon, SE1 7EH, United Kingdom
Active, not recruiting
Academisch Medisch Centrum (AMC)AMSTERDAM, 1105 AZ, Netherlands
Completed
Erasmus Medisch CentrumROTTERDAM, 3015 CE, Netherlands
Completed
Universitair Medisch Centrum GroningenGRONINGEN, 9713 GZ, Netherlands
Completed
University Medical Center UtrechtUTRECHT, 3584 CX, Netherlands
Completed
Hôpital PontchaillouRENNES CEDEX, 35033, France
Withdrawn
Hôpital de la Timone - MarseilleMARSEILLE, 13005, France
Active, not recruiting
Hopital Necker les enfants malades - ParisPARIS, 75015, France
Withdrawn
Hôpital Calmette - Lille CedexLILLE CEDEX, 59037, France
Withdrawn
Hospital Regional de MálagaMálaga, 29010, Spain
Withdrawn
Hospital Central de AsturiasOviedo, 33011, Spain
Withdrawn
Hospital de la Santa Creu i Sant Pau | Gynecology DepartmentBarcelona, 08041, Spain
Withdrawn
Ciutat Sanitaria i Universitaria de la Vall d'HebronBarcelona, 08035, Spain
Withdrawn
Hospital Universitario La Paz | IdiPAZ - Neurology DepartmentMadrid, 28046, Spain
Withdrawn
Hospital Universitari i Politècnic La FeValencia, 46026, Spain
Withdrawn
University Hospitals Cleveland Medical CenterCleveland, 44106, United States
Active, not recruiting
University of Wisconsin - MadisonMadison, 53792, United States
Withdrawn
Loma Linda Children's HospitalSan 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 interest
    date_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

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

Additional Information

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