Figure 1.
Study schematic of the open-label, phase 2/3 trial of human Glu-plasminogen in children and adults with congenital plasminogen deficiency. This is an ongoing open-label study with a 21-day screening period (for eligibility) and 3 treatment segments. Patients were given human Glu-plasminogen at 6.6 mg/kg administered as a 10- to 30-minute IV infusion every second, third, or fourth day based upon individual PK in segments 1 and 2. In segment 3, patients started with the same dose and frequency as in segment 2, with the investigator having the option to modify the dosing schedule based on clinical response and plasminogen activity trough levels. Patients will be treated for 48 weeks in Norway or until product licensing or study termination by the sponsor (whichever is later) in the United States.