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"
NCT ID: NCT03588299
Last Updated: 2025-12-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
11 participants
INTERVENTIONAL
2018-11-07
2026-11-03
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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BAY2599023 / (DTX201)
Adult patients with severe hemophilia A, who have been previously treated with FVIII products
BAY2599023 (DTX201)
Single escalating doses with 4 dose steps; Single intravenous (IV) administration.
Interventions
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BAY2599023 (DTX201)
Single escalating doses with 4 dose steps; Single intravenous (IV) administration.
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
18 Years
MALE
No
Sponsors
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Ultragenix pharmaceutical
UNKNOWN
Bayer
INDUSTRY
Responsible Party
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Locations
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Arkansas Children's Hospital - Hematology / Oncology
Little Rock, Arkansas, United States
C.S. Mott Children's Hospital - Hematology / Oncology
Ann Arbor, Michigan, United States
UW Health Carbone Cancer Center
Madison, Wisconsin, United States
SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
Sofia, , Bulgaria
APHP-Hopital Necker Enfants malades
Paris, , France
CHU Rennes - Hopital Pontchaillou
Rennes, , France
Universitätsklinikum des Saarlandes
Homburg, Saarland, Germany
Vivantes Klinikum im Friedrichshain
Berlin, , Germany
Academisch Medisch Centrum (AMC)
Amsterdam, , Netherlands
Universitair Medisch Centrum Groningen
Groningen, , Netherlands
Erasmus Medisch Centrum
Rotterdam, , Netherlands
University Medical Center Utrecht
Utrecht, , Netherlands
Manchester Royal Infirmary
Manchester, Greater Manchester, United Kingdom
Countries
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Related Links
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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.
Other Identifiers
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2023-505827-29-00
Identifier Type: OTHER
Identifier Source: secondary_id
2017-000806-39
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
19429
Identifier Type: -
Identifier Source: org_study_id