NCT03588299View on ClinicalTrials.gov

"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"

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

11

Participants

Timeline

Start Date

November 7, 2018

Primary Completion Date

November 3, 2026

Study Completion Date

November 3, 2026

Conditions
Hemophilia A
Interventions
DRUG

BAY2599023 (DTX201)

Single escalating doses with 4 dose steps; Single intravenous (IV) administration.

Trial Locations (13)

1756

SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD, Sofia

10249

Vivantes Klinikum im Friedrichshain, Berlin

35033

CHU Rennes - Hopital Pontchaillou, Rennes

48109

C.S. Mott Children's Hospital - Hematology / Oncology, Ann Arbor

53792

University of Wisconsin - Madison, Madison

66421

Universitätsklinikum des Saarlandes, Homburg

72202

Arkansas Children's Hospital - Hematology / Oncology, Little Rock

75015

APHP-Hopital Necker Enfants malades, Paris

1105 AZ

Academisch Medisch Centrum (AMC), Amsterdam

9713 GZ

Universitair Medisch Centrum Groningen, Groningen

3015 CE

Erasmus Medisch Centrum, Rotterdam

3584 CX

University Medical Center Utrecht, Utrecht

M13 9WL

Manchester Royal Infirmary, Manchester

Sponsors

Lead Sponsor

Bayer
All Listed Sponsors
collaborator

Ultragenix pharmaceutical

UNKNOWN

lead

Bayer

INDUSTRY

NCT03588299 - "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" | Biotech Hunter | Biotech Hunter