Efanesoctocog alfa treatment in patients with congenital hemophilia A suffering from inflammation of joint lining

Prior treatment

• Acute hemarthrosis at time of screening (clinical or ultrasound detected) or within the past 4 weeks before screening (clinical)
• Use of anticoagulant or antiplatelet drugs at the time of screening
• Participation in another clinical interventional trial in the 3 months before screening
• Current factor VIII inhibitor ≥0.6 BU/mL
• Current immune tolerance therapy
• Radiosynovectomy (RSO) or orthopedic surgery during the past 3 months or planned within the next 12 months

Other illness

• Known bleeding disorder other than hemophilia A
• Hypersensitivity to the active substance or to any of the excipients of the IMP
• History of thrombosis, myocardial infarction, other clinically relevant vascular diseases, or atrial fibrillation, or combination of risk factors that would significantly increase the cardiovascular risk during ERT or standard of care (SOC)

Other

• Life expectancy <12 months at the time of screening
• Patients of Asian ethnicity
• Close affiliation with the investigator (e.g. a close relative) or persons working at the study site, the sponsor or involved CRO
• Criteria which in the opinion of the investigator preclude participation for scientific reasons, for reasons of compliance, or for reasons of the patient’s safety
• Patient is in custody by virtue of an order issued either by the judicial or the administrative authorities
• Planned intensification of FVIII prophylaxis above the allowed doses specified in the protocol (Table 1, section 5.1)

• Written informed consent must be obtained before any study-specific tests or procedures are performed
• Male patients aged between 18 and 70 years at the first screening visit
• Patients must be capable of giving informed consent and have the ability to understand and follow study-related instructions
• Patients with severe to moderate congenital hemophilia A
• Regular prophylaxis with licensed FVIII products (SHL-FVIII, EHL-FVIII or HS-FVIII) at its recommended regimen during the last 6 months
• Synovitis of elbow, knee, and/or ankle joint (score ≥ 1 for synovial hypertrophy in HEAD-US) confirmed by Blinded ultrasound examination (BLUE) at the time of screening