Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

Study Identifier:
LTE15174
ClinicalTrials.gov Identifier:
EudraCT Identifier:
Sponsor:
Genzyme, a Sanofi Company
Collaborator:
N/A
Study Contact Information:
N/A
Unmapped

Study Details

Medical Condition
  • Hemophilia
Study Drug
  • Drug: Fitusiran
Date
Jan 2019 - Nov 2026
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Male
Age: 12+ 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
Phoenix Childrens Hospital Site Number : 8400009
Phoenix, Arizona, United States, 85016
Status
N/A
Location
Children's Hospital Los Angeles Site Number : 8400019
Los Angeles, California, United States, 90027
Status
N/A
Location
Center for Inherited Blood Disorders (CIBD) Site Number : 8400016
Orange, California, United States, 92868
Status
N/A
Location
Nemours Children's Clinic Site Number : 8400008
Jacksonville, Florida, United States, 32207
Status
N/A
Location
St Joseph's Children's Hospital of Tampa Site Number : 8400002
Tampa, Florida, United States, 33607
Status
N/A
Location
Rush University Medical Center -1725 W Harrison St Site Number : 8400001
Chicago, Illinois, United States, 60612-3863
Status
N/A