Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
Study Details
- Ignored
- Hemophilia A
- Hemophilia
- Drug: Standard of Care FVIII Replacement therapy
- Able and willing to provide written informed consent.
- Males ≥18 years of age.
- Clinically severe hemophilia A
- Previous exposure to FVIII therapy
- No prior history of hypersensitivity or anaphylaxis associated with an FVIII or intravenous immunoglobulin administration.
- No measurable inhibitor against FVIII
- Willing to participate and receive treatment in a future Spark hemophilia A gene therapy study.
- Documented active hepatitis B or C within the past 12 months of Screening
- Currently on antiviral therapy to treat hepatitis B or C;
- Documented significant liver disease within the past 6 months of Screening
- Have serological evidence of HIV-1 or HIV-2
- Anti-AAV-Spark 200 neutralizing titers ≥1:1
- Previously received SPK-8011;
- Previously dosed with any investigational or approved gene therapy product at any time or treated with an investigational drug within the last 12 weeks;
- Planned surgical procedure in the next 12 months requiring FVIII prophylactic treatment.
- Any history of chronic infection or other chronic disease, concurrent clinically significant major disease (such as liver abnormalities or type I diabetes) including active malignancy, except for non-melanoma skin cancer, any other condition or any other unspecified reasons that, in opinion of the Investigator or Sponsor, makes the participant unsuitable for participation and dosing in a future clinical study for Spark's hemophilia A gene therapy.
- Unable or unwilling to comply with the schedule of visits and/or study assessments described in the 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.