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Trial record 1 of 109 for:    Recruiting, Not yet recruiting, Available Studies | "Hemophilia A"
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Factor VIII Gene Therapy Study in Patients With Hemophilia A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03588299
Recruitment Status : Recruiting
First Posted : July 17, 2018
Last Update Posted : January 7, 2020
Ultragenyx Pharmaceutical Inc
Information provided by (Responsible Party):

Brief Summary:
The purpose of this study is to determine the safety and tolerability of the factor VIII gene transfer treatment with BAY 2599023 (DTX201) in individuals with severe hemophilia A.

Condition or disease Intervention/treatment Phase
Hemophilia A Drug: BAY2599023 (DTX201) Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : November 7, 2018
Estimated Primary Completion Date : March 16, 2021
Estimated Study Completion Date : July 22, 2025

Arm Intervention/treatment
Experimental: BAY2599023 / (DTX201)
Adult patients with severe hemophilia A, who have been previously treated with FVIII products
Drug: BAY2599023 (DTX201)
Single escalating doses with 3 dose steps; Single intravenous (IV) administration.

Primary Outcome Measures :
  1. Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interest [ Time Frame: Up to 52 weeks ]

Secondary Outcome Measures :
  1. Expression pattern of FVIII activity. [ Time Frame: Up to 5 years ]
    Determined using both a one-stage assay and chromogenic assay.

  2. 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 [ Time Frame: Up to 5 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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
  • Current evidence of measurable inhibitor against factor VIII, as assessed by the central laboratory and/or prior history of inhibitors to FVIII protein
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03588299

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Contact: Bayer Clinical Trials Contact (+) 1-888-8422937
Contact: For trial location information (Phone Menu Options '3' or '4') (+)1-888-84 22937

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Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc

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Responsible Party: Bayer Identifier: NCT03588299    
Other Study ID Numbers: 19429
2017-000806-39 ( EudraCT Number )
First Posted: July 17, 2018    Key Record Dates
Last Update Posted: January 7, 2020
Last Verified: January 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Bayer:
Severe hemophilia A (<1% FVIII activity)
Factor VIII gene therapy
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII