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Rtx001

A groundbreaking clinical trial is underway to evaluate RTX001, an innovative autologous engineered macrophage cell therapy, for patients with end-stage liver disease. This study aims to assess the safety, tolerability, and effectiveness of RTX001 in individuals who have recently experienced hepatic decompensation. The trial represents a significant step forward in the search for new treatments for liver cirrhosis and its complications.

Table of Contents

What is RTX001?

RTX001 is an innovative medical treatment being studied for patients with decompensated liver cirrhosis. It is classified as an autologous macrophage therapy, which means it uses the patient’s own immune cells to potentially treat their condition[1]. RTX001 is administered as a dispersion for infusion, which is a liquid form of the medication that is given directly into the bloodstream through an intravenous (IV) line[1].

Medical Condition: Decompensated Liver Cirrhosis

Liver cirrhosis is a condition where the liver becomes scarred and damaged over time. When cirrhosis progresses to the point where the liver can no longer function properly, it is called decompensated liver cirrhosis. This advanced stage of liver disease can lead to serious complications such as:[1]

  • Ascites: A buildup of fluid in the abdomen
  • Hepatic encephalopathy: Confusion and altered mental state due to toxins affecting the brain
  • Variceal bleeding: Bleeding from enlarged veins in the esophagus or stomach
  • HRS-AKI: Hepatorenal syndrome – acute kidney injury, a type of kidney failure associated with severe liver disease
  • SBP: Spontaneous bacterial peritonitis, an infection of the fluid in the abdominal cavity

How RTX001 Works

RTX001 is a type of cell therapy that uses macrophages, which are important cells in our immune system. Here’s how it works:[1]

  1. The patient’s own blood cells are collected through a process called leukapheresis.
  2. These cells are then modified in a laboratory to become specialized macrophages (RTX001).
  3. The modified cells are then given back to the patient through an IV infusion.

The goal of this treatment is to potentially help improve liver function and reduce the complications associated with decompensated liver cirrhosis.

Clinical Trial Details

RTX001 is currently being studied in a clinical trial called EMERALD. This is an open-label Phase 1/2 multicentre study, which means that both the researchers and participants know which treatment is being given, and it’s being conducted at multiple hospitals or medical centers[1].

The study is designed to evaluate the safety, tolerability, and efficacy of RTX001 in patients with decompensated liver cirrhosis. It’s important to note that this is an experimental treatment, and its effectiveness and safety are still being determined[1].

Eligibility Criteria

To participate in this study, patients must meet certain criteria. Some key eligibility requirements include:[1]

  • Age: 18 to 75 years old
  • Diagnosed with liver cirrhosis
  • Recent hospitalization for a major hepatic decompensation event
  • MELD score (a measure of liver disease severity) between 12 and 20
  • No recent alcohol misuse

There are also several exclusion criteria, such as certain co-existing medical conditions or recent use of other experimental treatments. A healthcare provider can provide more detailed information about eligibility[1].

Study Objectives

The main goals of this study are:[1]

  1. To evaluate the safety and tolerability of RTX001
  2. To assess the clinical efficacy of RTX001
  3. To measure changes in MELD scores (an indicator of liver disease severity)
  4. To compare and evaluate the characteristics and health issues of participants after treatment

Safety and Efficacy Measures

The study will closely monitor participants for any side effects or adverse events. It will also look at several measures to determine if RTX001 is effective, including:[1]

  • Time to and occurrence of additional liver-related complications
  • Changes in MELD scores
  • Survival rates and need for liver transplantation
  • Overall improvement in liver function and related symptoms

Treatment Process

The study is divided into four parts:[1]

  1. Screening and Stabilization: Participants are evaluated to ensure they meet the study criteria.
  2. Cell Collection and RTX001 Manufacture: Participants receive a medication called filgrastim to increase the number of certain blood cells. Then, these cells are collected through leukapheresis and used to create RTX001.
  3. Treatment Phase: Eligible participants receive RTX001 infusions.
  4. Long-term Follow-up: Participants are monitored for 2 years after their final dose of RTX001 to assess long-term safety and efficacy.

It’s important to remember that participating in a clinical trial is a significant decision that should be made in consultation with your healthcare provider. While RTX001 shows promise, it is still an experimental treatment, and more research is needed to fully understand its benefits and risks[1].

Aspect Details
Study Type Phase 1/2 open-label clinical trial
Treatment RTX001 (autologous engineered macrophage cell therapy)
Target Condition End-stage liver disease with recent hepatic decompensation
Administration Intravenous infusion (up to four doses)
Primary Outcomes Safety, tolerability, adverse events, infusion reactions
Secondary Outcomes Time to clinical events, mortality, changes in MELD3.0 score
Participant Groups Stabilised Group and Subsequent Decompensation Group
Control Data Compared to external control data from OPAL Natural History Study

FAQ

  • What is RTX001? RTX001 is an autologous engineered macrophage cell therapy. It's made from a patient's own white blood cells, specifically macrophages, which are modified to have anti-inflammatory and anti-fibrotic effects. This therapy is designed to help improve liver function in people with cirrhosis.
  • How is RTX001 produced? RTX001 is produced by first collecting white blood cells from the patient through a process called leukapheresis. These cells are then sent to a manufacturing facility where they are engineered to become RTX001. The final product is then given back to the same patient who donated the cells.
  • Who is eligible for this clinical trial? The trial is designed for patients with end-stage liver disease who have recovered from a recent hepatic decompensation event within the past 6 months. Participants are divided into two groups: those who remain clinically stable and those who have had a further decompensation event.
  • How is RTX001 administered? RTX001 is administered through intravenous infusion. Participants may receive up to four doses of RTX001 during the course of the study.
  • What are the main outcomes being measured in this trial? The primary outcomes focus on safety and tolerability, including the incidence and severity of treatment-emergent adverse events and infusion reactions. Secondary outcomes include time to clinical events (such as hepatic decompensation or liver transplantation), mortality, and changes in the Model for End Stage Liver Disease (MELD3.0) score.

Ongoing Clinical Trials on Rtx001

  • Safety and efficacy study of RTX001 autologous macrophage therapy in patients with decompensated liver cirrhosis

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Spain

Glossary

  • Autologous: Derived from the same individual. In this context, it means the therapy is made from and given back to the same patient.
  • Macrophages: A type of white blood cell that plays important roles in the immune system, including fighting infections and promoting tissue repair.
  • Liver Cirrhosis: A late stage of scarring (fibrosis) of the liver caused by many forms of liver diseases and conditions.
  • Hepatic Decompensation: A serious complication of liver cirrhosis where the liver can no longer perform its vital functions effectively.
  • Leukapheresis: A medical procedure in which white blood cells are separated from the blood for collection.
  • Anti-inflammatory: Reducing inflammation or swelling in the body.
  • Anti-fibrotic: Preventing or reducing the formation of excess fibrous tissue in organs.
  • MELD3.0 Score: Model for End-Stage Liver Disease, a scoring system used to assess the severity of chronic liver disease and prioritize patients for liver transplantation.
  • Intravenous Infusion: A method of delivering medications or fluids directly into a vein.
  • Treatment-emergent Adverse Events (TEAEs): Any unfavorable and unintended sign, symptom, or disease that appears or worsens after starting a medical treatment.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-efficacy-of-rtx001-and-filgrastim-for-patients-with-decompensated-liver-cirrhosis/