Defeat Rare Disease in Asia and Pacific (APAC) DRDA - Haemophilia
Study Details
- Hemophilia
- For Experienced/Senior Treating Physicians
- Experienced/senior haematologist treating haemophilia (adult or paediatric) or physician treating patients with haemophilia. (PWH), with greater than 5 years of experience.
- Able to communicate in English or country specific language.
- Agree/sign informed consent before data collection. For Patients Organisations
- Patient organisation supporting haemophilia.
- Expert having greater than 3 years of work experience with the patient organisation.
- Expert level understanding of haemophilia landscape and care pathway in the relevant country.
- Able to communicate in English or country specific language.
- Agree/sign informed consent before data collection. For Patients/Caregivers
- Patients of all age groups, diagnosed with haemophilia A or haemophilia B (with or without inhibitors) for greater than 2 years.
- For patients less than 18 years of age (\*less than 19 years for South Korea), their caregivers aged greater than or equal to 18 years (\*greater than or equal to19 years for South Korea) should sign the informed consent.
- \*For South Korea, the adult age is 19 years.
- Able to communicate in English or country specific language.
- Agree/sign informed consent before data collection.
- There is no explicit exclusion criterion in this study, thus any participants group who answers "no" to any of the inclusion criteria will be excluded from the study.
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.