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"

Study Identifier:
19429
ClinicalTrials.gov Identifier:
EudraCT Identifier:
Sponsor:
Bayer
Collaborator:
Ultragenix pharmaceutical
Study Contact Information:
N/A
Unmapped

Study Details

Medical Condition
  • Hemophilia A
Study Drug
  • Drug: BAY2599023 (DTX201)
Date
Nov 2018 - Nov 2026
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Male
Age: 18+ years years
Requirements Information

Protocol Summary

This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

Trial Locations

Location
Status
Location
Arkansas Children's Hospital - Hematology / Oncology
Little Rock, Arkansas, United States, 72202
Status
N/A
Location
C.S. Mott Children's Hospital - Hematology / Oncology
Ann Arbor, Michigan, United States, 48109
Status
N/A
Location
University of Wisconsin - Madison
Madison, Wisconsin, United States, 53792
Status
N/A
Location
SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
Sofia, Bulgaria, 1756
Status
N/A
Location
APHP-Hopital Necker Enfants malades
Paris, France, 75015
Status
N/A
Location
CHU Rennes - Hopital Pontchaillou
Rennes Cedex, France, 35033
Status
N/A