A Phase II, Prospective, Open Label, Standard of Care (SOC) Controlled, Study to Evaluate the Safety and Efficacy of Glassia® in Lung Transplantation during the First 6 Months

Purpose AAT has been reported to have anti-apoptotic and anti-inflammatory effects in addition to the canonical anti protease activity. This study aimed to evaluate the safety and efficacy of human, α-1 antitrypsin (AAT) [GLASSIA®] add-on pharmacotherapy in the treatment of patients undergoing first lung transplantation. Methods Thirty patients aged 18 and above (Table 1) were randomized 2:1 to receive SOC or SOC with intravenous AAT at the time of the lung transplant as a loading dose (90 mg/kg), and then 5 doses (30 mg/kg) every 2 days, followed by 3 biweekly (120 mg/kg), and 10 monthly (240 mg/kg) doses to 48 weeks. The following parameters were monitored: PGD, acute rejection, pulmonary function, and adverse events. Results We report here the short term (6 month) outcomes as part of an interim analysis. Patient demographics are shown in Table 1. AAT was associated with a trend to an increase in PaO 2 /FiO 2 and a decrease in PGD grade at day 3, and a decrease in the duration of mechanical ventilation after surgery. Acute rejection was observed in 4 AAT patients (21%) vs 3 SOC only patients (30%) and pulmonary infections were observed in 10 AAT (53%) vs 5 events in 4 (40%) SOC only patients. There were no adverse events that were considered to be related to study drug. Mortality was similar between arms and all the events (6) were related to sepsis. Conclusion AAT treatment is safe and might improve PGD characteristics and mechanical ventilation duration after lung transplantation.