A Proof-of-concept Study to Reduce Treatment Burden in Haemophilia Participants Receiving Factor VIII and Factor IX Infusions
Study Details
- Hemophilia
- Inclusion criteria:
- Male participant over the age of 6 years, with diagnosed Congenital Hemophilia A or B, whatever the severity is
- Participant under long-term prophylaxis with intravenous Factor VIII or Factor IX infusions
- Participant (or the legal guardians if participant age <18 years) able and willing to give written informed consent and to comply with the requirements of the study protocol
- Participant affiliated to the national social security or beneficiary to such insurance
- Exclusion criteria:
- Participant with known or suspected hypersensitivity to virtual-reality based tools
- Participant with central venous line for the administration of Factor VIII or Factor IX
- Participant (and the legal guardians if participant age <18 years) with history of unreliability or non-cooperation (including for completion of self-reported questionnaires)
- Participant (and the legal guardians if participant age <18 years) with insufficient comprehension of French language
- Participant taking part in another clinical trial
- Participant deprived of his liberty by judicial or administrative order
- NB: participants with haemophilia A having participated in the test phase of the study could be included in the clinical study itself after a period of 4 months minimum with no use of the virtual-reality based solution.
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.