A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
Study Details
- Hemophilia A
- Drug: Fitusiran (SAR439774)
- Biological: Clotting factor concentrates (CFC) or bypassing agents (BPA)
- Biological: Antithrombin concentrate (ATIIIC)
- Biological: Emicizumab
- Male participants must be ≥12 years of age inclusive, at the time of signing the informed consent
- Diagnosis of severe congenital hemophilia A (FVIII \< 1%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.
- Inhibitor titer of ≥0.6 BU/mL at Screening, or
- Inhibitor titer of \<0.6 BU/mL at Screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, or
- Inhibitor titer of \<0.6 BU/mL at Screening with medical record evidence of anamnestic response.
- Participants who are currently on the full labeled dose of emicizumab prophylaxis, irrespective of inhibitor/non-inhibitor status.
- Signed informed consent/assent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- Participants are excluded from the study if any of the following criteria apply:
- Known coexisting bleeding disorders
- History of antiphospholipid antibody syndrome.
- History of arterial or venous thromboembolism, atrial fibrillation, significant valvular disease, myocardial infarction, angina, transient ischemic attack, or stroke. Participants who have experienced thrombosis associated with indwelling venous access may be enrolled.
- Presence of clinically significant liver disease
- Current or prior participation in a fitusiran trial
- Current or prior participation in a gene therapy trial
- AT activity \<60% at Screening, as determined by central laboratory measurement
- Coexisting thrombophilic disorder - Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis
- Presence of acute hepatitis, ie, hepatitis A, hepatitis E.
- Presence of acute or chronic hepatitis B infection
- Known to be HIV positive with CD4 count \<200 cells/μL.
- Reduced renal function
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
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.