Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC) administration provides for treatment options in the absence of reliable vascular access, increased convenience and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes.
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Annualized bleeding rate (ABR) of treated bleeding events
Timeframe: Through Observational Phase (6months) and Active Treatment Phase (12 months) for total of approximately 18 months
Incidence and severity of thrombotic events
Timeframe: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months
Incidence of anti drug antibody [ADA] against PF-06741086
Timeframe: Throughout Active Treatment Phase (12 months)
Incidence of clinically significant persistent neutralizing antibody [NAb] against PF-06741086
Timeframe: Throughout Active Treatment Phase (12 months)
Incidence and severity of injection site reaction
Timeframe: Throughout Active Treatment Phase (12 months)
Number of participants with clinically significant changes from baseline in physical exam
Timeframe: From Baseline through Observation and Active Treatment (approximately 18 months)
Incidence of clinically significant laboratory value abnormalities
Timeframe: From Screening through Observation and Active Treatment (approximately 18 months)
Incidence of severe hypersensitivity and anaphylactic reactions
Timeframe: From Screening through Observational and Active Treatment (approximately 18 months)
Incidence of adverse events and serious adverse events
Timeframe: From screening through Observation and Active treatment (approximately 18 months)
Number of participants with clinically significant changes from baseline in vital signs
Timeframe: From Baseline through Observation and Active Treatment (approximately 18 months)
Incidence and severity of thromboticangiopathy
Timeframe: Throughout Active Treatment Phase (12 months)
Incidence of intravascular coagulation/consumption coagulopathy
Timeframe: Throughout Active Treatment Phase (12 months)