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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".

Primary Purpose

Hemophilia A

Status
Active
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
BAY2599023 (DTX201)
Sponsored by
Bayer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring Severe hemophilia A (<1% FVIII activity), Factor VIII gene therapy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • Arkansas Children's Hospital
  • C.S. Mott Children's Hospital
  • University of Wisconsin - Madison
  • SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
  • Hopital Necker les enfants malades - Paris
  • Hôpital Pontchaillou
  • Universitätsklinikum des Saarlandes
  • Vivantes Klinikum im Friedrichshain
  • Academisch Medisch Centrum (AMC)
  • Universitair Medisch Centrum Groningen
  • Erasmus Medisch Centrum
  • University Medical Center Utrecht
  • Manchester Royal Infirmary

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BAY2599023 / (DTX201)

Arm Description

Adult patients with severe hemophilia A, who have been previously treated with FVIII products

Outcomes

Primary Outcome Measures

Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interest

Secondary Outcome Measures

Expression pattern of FVIII activity.
Determined using both a one-stage assay and chromogenic assay.
Proportion of patients in the respective dose step, that reached an expression of FVIII above 5%

Full Information

First Posted
July 6, 2018
Last Updated
October 10, 2023
Sponsor
Bayer
Collaborators
Ultragenix pharmaceutical
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1. Study Identification

Unique Protocol Identification Number
NCT03588299
Brief Title
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".
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
November 7, 2018 (Actual)
Primary Completion Date
November 3, 2026 (Anticipated)
Study Completion Date
November 30, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bayer
Collaborators
Ultragenix pharmaceutical

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
Keywords
Severe hemophilia A (<1% FVIII activity), Factor VIII gene therapy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
BAY2599023 / (DTX201)
Arm Type
Experimental
Arm Description
Adult patients with severe hemophilia A, who have been previously treated with FVIII products
Intervention Type
Drug
Intervention Name(s)
BAY2599023 (DTX201)
Intervention Description
Single escalating doses with 4 dose steps; Single intravenous (IV) administration.
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
Expression pattern of FVIII activity.
Description
Determined using both a one-stage assay and chromogenic assay.
Time Frame
Up to 5 years
Title
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

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202-3500
Country
United States
Facility Name
C.S. Mott Children's Hospital
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
University of Wisconsin - Madison
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
City
Sofia
ZIP/Postal Code
1756
Country
Bulgaria
Facility Name
Hopital Necker les enfants malades - Paris
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Hôpital Pontchaillou
City
Rennes Cedex
ZIP/Postal Code
35033
Country
France
Facility Name
Universitätsklinikum des Saarlandes
City
Homburg
State/Province
Saarland
ZIP/Postal Code
66421
Country
Germany
Facility Name
Vivantes Klinikum im Friedrichshain
City
Berlin
ZIP/Postal Code
10249
Country
Germany
Facility Name
Academisch Medisch Centrum (AMC)
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
Facility Name
Universitair Medisch Centrum Groningen
City
Groningen
ZIP/Postal Code
9713 GZ
Country
Netherlands
Facility Name
Erasmus Medisch Centrum
City
Rotterdam
ZIP/Postal Code
3015 CE
Country
Netherlands
Facility Name
University Medical Center Utrecht
City
Utrecht
ZIP/Postal Code
3584 CX
Country
Netherlands
Facility Name
Manchester Royal Infirmary
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing 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.
Links:
URL
https://clinicaltrials.bayer.com/
Description
Click here to find information for studies related to Bayer products. To find this study enter the ClinicalTrials.gov identifier (NCT) number or Bayer Study Identifier (ID) in the search field.

Learn more about this trial

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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