Phase 12 Study of RTX001 Autologous Macrophage Therapy for Adults with Liver Cirrhosis and Recent Liver Decompensation

What this Trial Is About

Researchers are evaluating the safety and effectiveness of RTX001, an autologous engineered macrophage cell therapy, in patients with end-stage liver disease who have recently experienced hepatic decompensation. This first-in-human Phase 12 open-label study aims to understand how RTX001, made from a persons own white blood cells, affects liver function and disease progression compared to data from a similar patient population in an external natural history study. Participants will undergo leukapheresis to collect white blood cells, which are then used to manufacture RTX001. After manufacturing, participants are assigned to one of two groups based on their current liver stability those who are stable and those who have had another decompensation event but have since stabilized. Both groups will receive up to four intravenous infusions of RTX001. Treatment timing depends on the participants clinical status. During the study, participants will be monitored closely for safety and tolerability at each infusion and up to two weeks afterwards, with follow-up continuing for 2.5 years. Researchers will assess clinical events, mortality, and changes in liver disease severity using the Model for End Stage Liver Disease score. Participants will have regular evaluations including medical assessments and laboratory tests throughout the study to track the therapys impact and ensure safety.

RTX001 Autologous Engineered Macrophages for Liver Cirrhosis