Haemophilia and Bone Loss - PHILEOS Study
Study Details
- Hemophilia
- Biological: Blood sampling for patients only
- Healthy Volunteers :
- Healthy men aged between 20 to 60 years old
- Haemophilic Patients:
- Haemophilia A and B patients, irrespective of the disease form (mild, moderate, severe with or without prophylaxis)
- Haemophilic patients aged between 20 to 60 years old
- Severe Haemophilia A patients with prophylaxis : last factor VIII injection more than 48 to 120 hours (depending on on the prophylactic treatment) prior blood sampling dedicated to the this research
- Severe Haemophilia B patients : last factor IX injection more than 5 to 21 days (depending on the prophylactic treatment) prior blood sampling dedicated to the this research
- Healthy Volunteers:
- History of disease known to influence bone metabolism (hyperthyroidism, hyperparathyroidism, hypercorticism, hypogonadism, diseases that require long-term use of corticoids, …)
- Past or present treatment with any osteoporotic medication other than Vit D or Ca++
- Presence of two total hip prostheses
- HIV documented infection
- HCV documented infection (in progress or cured) at cirrhotic stage
- Haemophilic Patients:
- Haemophilic patients with current or history of inhibitor anti-fVIII or anti-fIX (\>5 Bethesda Units)
- Treatment with HEMLIBRA (Emicizumab). Unless it is possible to use a result of thrombin generation prior to this treatment and achieved with a residual rate not greater than or equal to 5%.
- History of disease known to influence bone metabolism and not related to haemophilia (hyperthyroidism, hyperparathyroidism, hypercorticism, hypogonadism, diseases that require long-term use of corticoids, …)
- Past or present treatment with any anti-osteoporotic medication other than Vit D or Ca++
- Presence of two total hip prostheses
- HIV documented infection
- HCV documented infection (in progress or cured) at cirrhotic stage
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.