Trial status: actively recruiting

Trial ID: NCT06823713

What is the EMERALD study?

EMERALD is a clinical trial designed to test if a new treatment called RTX001 is safe, easy to cope with, and works well for people suffering from decompensated liver cirrhosis. The acronym spells “Evaluating Macrophages Engineered to Resolve Advanced Liver Disease”.

Edinburgh Royal Infirmary is the first hospital to start the trial. In the coming months this will expand to include more hospitals across England. The trial is sponsored by a company called Resolution Therapeutics Ltd.

What is the study drug being tested?

RTX001 is a novel treatment made by converting a small volume of a person’s own white blood cells into a different type of cell called macrophages. Macrophages are naturally occurring cells from our immune system and play an important role in eliminating disease and damaged cells. The process of modifying a cell is known as ‘cell-engineering’ and is carried out in a specialist laboratory. RTX001 is also an autologous product which means it can only be given to the person from whom the blood cells were collected.

How can engineered macrophages help to treat liver cirrhosis?

Scientific studies have shown that macrophages could play a helpful role by reducing inflammation in the liver and helping the liver to heal itself. This is because macrophages play a part in removing liver damaged tissue and scars, stopping new scars from forming, and stimulating the regeneration of the liver. To boost these properties, researchers at Resolution Therapeutics have engineered the macrophages in a laboratory with the goal of giving them a stronger and longer-acting benefit for patients.

How are the white blood cells collected?

To produce RTX001, participants will first begin treatment with a medicine called filgrastim. This is an injection that will help increase the number of white blood cells. Participants have up to five daily injections, some of which may be taken at home. Following the last injection, the white blood cells will be collected through a vein using a process called leukapheresis. Leukapheresis is a procedure in which white blood cells are separated from the collected blood using a specific machine. A sample of these white blood cells  will then be used to make RTX001.

How is RTX001 treatment administered?

RTX001 is administered through a tube into a vein in an arm (this is called intravenous infusion). This takes about 30 minutes. Participants will receive up to 4 doses over the course of the study. One week after each dose, participants will have a visit at the hospital, and then a telephone call a week after that, to check for any side effects or any other problems.

How do I find out if I am eligible to take part in EMERALD?

Before joining the study, people with decompensated liver cirrhosis will be asked to undergo screening assessments at participating hospitals (see full list below). These assessments are to confirm a participant’s suitability to take part and include checking vital signs, blood samples and a physical exam. If after 6 weeks participants are considered clinically stable by the study doctor, they will be enrolled into EMERALD.

Taking part in EMERALD

Who can take part in EMERALD?

EMERALD is focused on people with “decompensated liver cirrhosis”. If you are interested in taking part in the study, potential study participants must meet certain criteria, such as:

• be aged 18-75 years old
• have been diagnosed with liver cirrhosis
• have recently been hospitalised for a serious liver-related problem and not had any similar problems in the last 6 months, or
• have a large amount of abdominal fluid (ascites) that needs to be drained multiple times despite trying medical and dietary treatments
• be willing and able to comply with study procedures, including limiting the amount of alcohol you drink to no more than 3 units of alcohol every day for women or more than 4 units per day for men

Not everyone with liver cirrhosis will be able to take part so the study doctor will check a participant’s medical history and other requirements during the screening assessments to confirm whether or not a participant is eligible to join the study.

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What will I have to do if I take part?

Participating in the study means more visits to the hospital in order to collect all the necessary data.

Here’s a summary of what a study participant can expect during their involvement in the study:

• Attend all study visits for the duration of the study
• Undergo safety tests which may involve blood tests, check of vital signs, tests on blood and urine samples, alcohol blood rests, liver scans and filling out of questionnaires
• Answer questions honestly about changes in health status, visits to other doctors or hospital admissions.
• Answer questions honestly about changes in medication use, including prescribed medications, over the counter medications, herbal remedies, and vitamins.
• Tell the study doctor if there is a chance of the participant /participants partner may be pregnant.
• Tell your study doctor if the participant changes their mind about taking part in the study or has any concerns.

All this takes place over two years. Participants are free to leave the study at any time without giving a reason. If a participant  leaves the study, they will not have the extra appointments, but will still receive all the normal care for treatment of their liver cirrhosis.

Are there any costs/payments for taking part in this study?

There will be no cost to participants for taking part in EMERALD. Participants will be able to claim money to cover travel expenses if needed for extra clinic visits that do not coincide with routine appointments.

Next Steps

How to get involved in EMERALD?

Edinburgh Royal Infirmary is the first hospital to start the trial, and in the coming months more hospitals across England will take part in the study.