A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
Study Details
- Hemophilia
- Drug: Fitusiran
- Drug: Clotting factor concentrates (CFC) or bypassing agents (BPA)
- Drug: Antithrombin concentrate (ATIIIC)
- Inclusion Criteria:
- Diagnosis of severe congenital hemophilia A or B (FVIII \<1% or FIX level ≤2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.
- For participants currently not on prophylaxis (CFC or BPA on-demand): A minimum of 4 bleeding episodes requiring BPA (inhibitor participants) or CFC (non-inhibitor participants) treatment within the last 6 months prior to screening.
- Willing and able to comply with the study requirements and to provide written informed consent and assent in the case of participants under the age of legal consent, per local and national requirements
- Exclusion Criteria:
- Known co-existing bleeding disorders other than congenital hemophilia A or B
- History of arterial or venous thromboembolism, not associated with an indwelling venous access
- History of intolerance to SC injection(s).
- Current participation in immune tolerance induction therapy (ITI)
- Prior gene therapy
- Current or prior participation in a fitusiran trial
- Current or prior participation in a gene therapy trial
- Received an investigational drug or device within 30 days prior to the screening visit or within 5 half-lives of the investigational drug (or device) prior to the screening visit, whichever is longer
- Presence of clinically significant liver disease AT activity \<60% at Screening
- Co-existing 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.