About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male or female age ≥18-75 years.
Patient confirms willingness/ability to comply with all study procedures.
Clinical and radiological features that correlate with a diagnosis of cirrhosis.
Transient elastography (Fibroscan) >15 kPa.
Disqualifiers
any viral hepatitidies, or
autoimmune and cholestatic aetiologies including, but not limited to, primary biliary cholangitis and primary sclerosing cholangitis.
Acute liver disease in the absence of underlying liver cirrhosis, including, but not limited to, drug induced liver injury.
Any current organ failure requiring more than outpatient supportive care, and not associated with the participant's qualifying hepatic decompensation event.
Trial design
Single group
Treatments tested in this trial
RTX001
DrugRTX001 is an autologous engineered regenerative macrophage cell therapy
Treatment groups
Trial outcomes
Primary outcomes
Safety and Tolerability
Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Safety and Tolerability
Incidence and severity of infusion reactions
Secondary outcomes
Time to clinical event
Time to clinical event, defined as all hepatic decompensation events including SBP and/or HRS-AKI, new listing for liver transplantation, liver transplantation, or death.
Time to mortality
Time-to all-cause mortality (hepatic related and all-cause).
Change in Model for End Stage Liver Disease score 3.0 (MELD3.0)
To evaluate changes in Model for End Stage Liver Disease (MELD3.0) scores, from baseline to end of study in the Stabilised Group participants.
Sponsors and contacts
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