Comparing Fitusiran with Clotting factor concentrates (CFC) or for hemophilia

Exclusion

• −History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
• −Presence of an active Hepatitis C virus infection
• −Presence of acute hepatitis A or Hepatitis E virus infection.
• −Presence of acute or chronic hepatitis B virus infection.
• −Human immunodeficiency virus (HIV) positive with a CD4 count of \<400 cells/μL.
• −Participants not previously exposed to fitusiran are excluded from the study if any of the following criteria apply:
• −Known co-existing bleeding disorders other than hemophilia A or B.
• −Presence of clinically significant liver disease.
• −History of antiphospholipid antibody syndrome.
• −History of arterial or venous thromboembolism, unrelated to an indwelling venous access
• −Any condition (eg, medical concern), which in the opinion of the InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →, would make the participant unsuitable for dosing or which could interfere with the study compliance, the participant's safety and/or the participant's participation in the completion of the treatment period of the study.
• −Subjects with a central or peripheral indwelling catheter, with a history of venous access complications (such as infections, thrombosis) leading to hospitalization and/or systemic anticoagulation therapy in the last 12 months.
• −Completion of a surgical procedure within 14 days prior to screeningScreeningThe checks done before joining, to see whether a study fits.Read more →, or currently receiving additional BPA infusion for postoperative hemostasis.
• −History of intolerance to SC injection(s).
• −Current participation in ITI therapy.
• −The use of emicizumab (Hemlibra®) or any non-factor bleed management treatment within 6 months prior to screening
• −Prior gene therapy
• −Current or future participation in another clinical study, scheduled to occur during this study, involving an investigational product other than fitusiran or an investigational device.
• −AT activity \<60% at screening, as determined by central laboratory analysis.
• −Co-existing thrombophilic disorder.
• −Platelet count ≤100 000/μL.
• −Presence of acute infection at screening.
• −Estimated glomerular filtration rate ≤45 mL/min/1.73 m2 (using the Schwartz formula).
• −The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trialInterventional studyA study where participants are given something to see what happens.Read more →.
• −At screening, anticipated need of surgery during the study or planned surgery scheduled to occur during the study.