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 : Recruiting
First Posted : July 17, 2018
Last Update Posted : November 3, 2020
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia A||Drug: BAY2599023 (DTX201)||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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 :||October 30, 2022|
|Estimated Study Completion Date :||November 30, 2026|
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.
- 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 ]
- Expression pattern of FVIII activity. [ Time Frame: Up to 5 years ]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% [ Time Frame: At 6 months and 12 months following the IV administration of BAY2599023 ]
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
|Contact: Bayer Clinical Trials Contact||(+) email@example.com|
|Contact: For trial location information (Phone Menu Options '3' or '4')||(+)1-888-84 22937|