Health-Related Quality of Life Outcomes and Bleeding Rates Among Patients With Severe Hemophilia A on Emicizumab
Study Details
- Hemophilia A
- Drug: Emicizumab
- Inclusion Criteria Part A: ABR
- Signed Informed Consent/Assent Form
- Age 4 years at time of signing Informed Consent Form
- Ability to comply with the study protocol, in the investigator's judgment
- Diagnosis of hemophilia A (any severity) on emicizumab or factor concentrate prophylaxis for at least 12 months each
- Plan to be adherent to emicizumab prophylaxis during the study
- Patient with inhibitors
- Patient without inhibitors Part B: HRQoL and Arthropathy
- Signed Informed Consent/Assent Form
- Age 4 years at time of signing Informed Consent Form
- Ability to comply with the study protocol, in the investigator's judgment
- Diagnosis of hemophilia A (any severity) who has decided to switch to emicizumab prophylaxis
- Plan to be adherent to emicizumab prophylaxis during the study
- Individuals of childbearing age will be included in this study. Their participation involves voluntary completion of a questionnaire. There are no additional precautions required for this population.
- Patient/parent must be able to read and write English/Spanish
- Exclusion Criteria:
- Part A: ABR
- Patients who are on a clinical trial for prophylaxis
- Patients with other bleeding disorders needing any scheduled treatment
- No eligibility restrictions will be based on gender, race, ethnic background or economic status
- Part B: HRQoL and Arthropathy
- Patients who are on a clinical trial for prophylaxis
- Patients with other bleeding disorders needing any scheduled treatment
- Patients/parents who refuse to complete questionnaire or have joint health assessment by HJHS
- No eligibility restrictions will be based on gender, race, ethnic background or economic status
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.