Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B.
Study Details
- Hemophilia
- Hemophilia B
- Drug: nonacog beta pegol
- Inclusion Criteria:
- Male patients with moderately severe or severe congenital haemophilia B with a Factor IX activity level below or equal to 2% according to medical records
- Age below or equal to 12 years (until patient turns 13 years, at time of inclusion)
- Body weight above or equal to 10 kg
- History of at least 50 exposure days (EDs) to other FIX products
- The patient and/or parent(s)/caregiver are capable of assessing a bleeding episode, keeping an electronic diary (eDiary), capable of conducting home treatment and otherwise able to follow trial procedures
- Exclusion Criteria:
- Known history of FIX inhibitors
- Current FIX inhibitors above or equal to 0.6 Bethesda Units (BU)
- Congenital or acquired coagulation disorder other than haemophilia B
- Platelet count below 50,000/mcL at screening
- Alanine aminotransferase (ALT) above 3 times the upper limit of normal reference ranges at screening
- Creatinine level above or equal to 1.5 times above the upper normal limit of normal reference ranges at screening
- Human immunodeficiency virus (HIV) positive, defined by medical records, and with a CD4+ lymphocyte count below or equal to 200/mcL
- Immune modulating or chemotherapeutic medication (except single pulse treatment, inhaled and topical steroids)
- Previous arterial thrombotic events (myocardial infarction and intracranial thrombosis, as defined by medical records)
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.