Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B

Study Identifier:
AGT4HB
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
Sponsor:
St. Jude Children's Research Hospital
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI),Hemophilia of Georgia, Inc.,Children's Hospital of Philadelphia,University College, London
Study Contact Information:
N/A
Unmapped

Study Details

Medical Condition
  • Hemophilia B
Study Drug
  • Drug: scAAV2/8-LP1-hFIXco
Date
Feb 2010 - Jun 2032
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
Stanford Medical School
Stanford, California, United States, 94305
Status
N/A
Location
University of Kentucky
Lexington, Kentucky, United States, 40536
Status
N/A
Location
Oregon Health and Science University
Portland, Oregon, United States, 97239
Status
N/A
Location
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States, 15213
Status
N/A
Location
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38119
Status
N/A
Location
University of Texas Southwestern
Dallas, Texas, United States, 75390-9063
Status
N/A