Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
Study Details
- Hemophilia A
- Inclusion Criteria
- Participants are eligible to be included in the study only if all of the following criteria apply:
- Have received a single intravenous administration of SPK-8011 or SPK-8016 in either Study SPK-8011-101 or SPK-8016-101, respectively.
- Understand the purpose and risks of the study and provide signed and dated informed consent before undergoing any study-specific procedures.
- Exclusion Criteria
- Participants are excluded from the study if any of the following criteria apply:
- Unable or unwilling to comply with the schedule of visits and study assessments as described in this study protocol.
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.