Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

Exclusion Criteria

• •Detectable pre-existing antibodies to the AAV5 capsid.
• •Any evidence of active infection or any immunosuppressive disorder, except for human immunodeficiency virus (HIV) infection. HIV-positive participants who meet all other eligibility criteria may be included.
• •Significant liver dysfunction
• •Most recent, prior FibroScan or liver biopsy showing significant fibrosis
• •Evidence of any bleeding disorder not related to HA.
• •Platelet count of \<100E9/L.
• •Creatinine ≥1.5 mg/dL.
• •Liver cirrhosis of any etiology as assessed by liver ultrasound.
• •Chronic or active hepatitis B
• •Active hepatitis C
• •Active malignancy, except non-melanoma skin cancer
• •History of hepatic malignancy
• •History of arterial or venous thromboembolic events
• •Known inherited or acquired thrombophilia, including conditions associated with increased thromboembolic risk, such as atrial fibrillation
• •Treatment with any investigational product (IP) within 30 days or 5 half-lives of the IP prior to the Screening period.
• •Any condition that, in the opinion of the investigator or sponsor would prevent the participant from fully complying with the requirements of the study
• •Prior treatment with any vector or gene transfer agent.
• •Major surgery planned in the 52-week period following the infusion with BMN 270.
• •Use of systemic immunosuppressive agents, not including corticosteroids, or live vaccines within 30 days before the BMN 270 infusion.
• •Concurrent enrollment in another clinical study unless it is an observational (non-interventional) clinical study that does not interfere with the requirements of the current protocol or have the potential to impact the evaluation of efficacy and safety of BMN 270 and with prior consultation with the medical monitor.
• •Known allergy or hypersensitivity to BMN 270 IP formulation.
• •Unwilling to receive blood or blood products for treatment of an AE and/or a bleeding episode.