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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".

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ClinicalTrials.gov Identifier: NCT03588299
Recruitment Status : Active, not recruiting
First Posted : July 17, 2018
Last Update Posted : January 6, 2023
Sponsor:
Collaborator:
Ultragenix pharmaceutical
Information provided by (Responsible Party):
Bayer

Study Description
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Brief 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.

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Allocation: N/A
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 : November 3, 2026
Estimated Study Completion Date : November 30, 2026

Resource links provided by the National Library of Medicine


Arms and Interventions
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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 4 dose steps; Single intravenous (IV) administration.


Outcome Measures
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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 5 years ]

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% [ Time Frame: At 6 months and 12 months following the IV administration of BAY2599023 ]

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

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
Contacts and Locations
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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 ClinicalTrials.gov identifier (NCT number): NCT03588299


Locations
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United States, Arkansas
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202-3500
United States, Michigan
C.S. Mott Children's Hospital
Ann Arbor, Michigan, United States, 48109
United States, Wisconsin
University of Wisconsin - Madison
Madison, Wisconsin, United States, 53792
Bulgaria
SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
Sofia, Bulgaria, 1756
France
Hopital Necker les enfants malades - Paris
Paris, France, 75015
Hôpital Pontchaillou
Rennes Cedex, France, 35033
Germany
Universitätsklinikum des Saarlandes
Homburg, Saarland, Germany, 66421
Vivantes Klinikum im Friedrichshain
Berlin, Germany, 10249
Netherlands
Academisch Medisch Centrum (AMC)
Amsterdam, Netherlands, 1105 AZ
Universitair Medisch Centrum Groningen
Groningen, Netherlands, 9713 GZ
Erasmus Medisch Centrum
Rotterdam, Netherlands, 3015 CE
University Medical Center Utrecht
Utrecht, Netherlands, 3584 CX
United Kingdom
Manchester Royal Infirmary
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Bayer
Ultragenix pharmaceutical
More Information
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Additional Information:

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Responsible Party: Bayer
ClinicalTrials.gov Identifier: NCT03588299    
Other Study ID Numbers: 19429
2017-000806-39 ( EudraCT Number )
First Posted: July 17, 2018    Key Record Dates
Last Update Posted: January 6, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description:

Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

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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