A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

Study Identifier:
C0371002
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
Sponsor:
Pfizer
Collaborator:
N/A
Study Contact Information:
N/A
Unmapped

Study Details

Medical Condition
  • Hemophilia B
Study Drug
  • Biological: PF-06838435/ fidanacogene elaparvovec
Date
Jul 2019 - Nov 2022
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Male
Age: 18 - 65 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
University of California, San Francisco - Clinical Research Center
San Francisco, California, United States, 94143
Status
N/A
Location
University of California, San Francisco - Outpatient Hematology Clinic
San Francisco, California, United States, 94143
Status
N/A
Location
Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States, 80045
Status
N/A
Location
Indiana Hemophilia & Thrombosis Center, Inc.
Indianapolis, Indiana, United States, 46260
Status
N/A
Location
Indiana Hemophilia and Thrombosis Center, Inc
Indianapolis, Indiana, United States, 46260
Status
N/A
Location
St. Vincent Hospital & Health Care Center, Inc.
Indianapolis, Indiana, United States, 46260
Status
N/A