NCT06823713

RTX001 Autologous Engineered Macrophages for Liver Cirrhosis

An Open-label Phase 1/2 Multicentre Study to Evaluate the Safety, Tolerability and Efficacy of RTX001 Autologous Macrophages in Participants With Liver Cirrhosis Who Have Hepatic Decompensation (EMERALD)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Resolution Therapeutics Limited · Network
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to assess the safety and efficacy of RTX001 in patients with end-stage liver disease. This study is the first time RTX001, a macrophage cell therapy engineered to have an anti-inflammatory and anti-fibrotic effect, will be given to humans.

Detailed description

EMERALD is a first-in-human Phase 1/2 open label study designed to evaluate the safety, tolerability and efficacy of RTX001 in patients with end-stage liver disease following recent hepatic decompensation. RTX001 is an autologous engineered macrophage cell therapy. It is made from a person's own cells. It uses a type of white blood cell called macrophages. These cells have been found to help improve liver function in participants with liver cirrhosis. To produce RTX001, each study participant must first undergo steps that will allow us to collect white blood cells using a process called leukapheresis. Leukapheresis is a procedure in which white blood cells are separated from the collected blood using a specific machine. The collected white blood cells are sent to a manufacturing facility to make RTX001. As RTX001 is an autologous product, this means that it can only be given back to the same participant who donated the cells. The term "autologous macrophage" used in the study title refers to white blood cells (macrophages) coming from the same person's body (autologous). The data in this study will be compared to the external control data from a Natural History Study called OPAL (NCT06380335) which is being conducted in a similar participant population from primarily the same study sites in the United Kingdom (UK) and EU.

Conditions

• End-stage Liver Disease (ESLD)
• Cirrhosis, Liver
• Cirrhosis, Decompensated
• Liver Diseases
• Fibrosis and Cirrhosis of Liver
• Decompensated Liver Cirrhosis
• Decompensated Cirrhosis
• Steatotic Liver Disease

Interventions

Type	Name	Description
DRUG	RTX001	RTX001 is an autologous engineered regenerative macrophage cell therapy

Timeline

• Start date: 2024-10-15
• Primary completion: 2028-08-29
• Completion: 2028-11-29
• First posted: 2025-02-12
• Last updated: 2026-01-28

Locations

14 sites across 2 countries: Spain, United Kingdom