RecruitingPhase IInterventional

A CAR T trial for amyloid light chain amyloidosis (AL Amyloid)

This research is looking into a new treatment for a rare disease called AL amyloidosis. In this condition, abnormal proteins build up in organs like the heart or kidneys, causing damage. Standard treatments include chemotherapy or stem cell transplants, but sometimes the disease returns or doesn't improve. This study uses a new approach called CAR T-cell therapy. It involves taking your own immune cells (T-cells), specially modifying them in a lab to recognise and attack the abnormal cells causing AL amyloidosis, and then giving them back to you. This Phase 1 trial aims to find out if this new therapy is safe and whether it can help control the disease in people who haven't responded well to other treatments.

At a glance

Status

Recruiting

Phase

Phase I

Sponsor

University College London Cancer Trials Centre

Enrolment target

Start

31 May 2025

Estimated completion

30 May 2027

AL Amyloid

What is this study about?

AL amyloidosis is a rare but serious condition. It happens when certain cells in your bone marrow, called plasma cells, start making abnormal pieces of protein. These pieces can then stick together and form deposits called amyloid in different parts of your body, including vital organs like your heart, kidneys, liver or nerves. Over time, these deposits can stop your organs from working properly, leading to various health problems. While there are existing treatments like chemotherapy, which aims to kill the abnormal plasma cells, sometimes these treatments don't work well enough, or the disease comes back.

This study is exploring a new type of treatment called CAR T-cell therapy. It's a cutting-edge approach that uses your own body's immune system to fight the disease. Researchers take a specific type of white blood cell, called a T-cell, from your blood. These T-cells are then sent to a lab where they are genetically modified. This modification teaches your T-cells to recognise and attack the abnormal plasma cells that are creating the harmful amyloid proteins. Once modified, these 'super-charged' T-cells are given back to you.

The researchers have developed two types of these CAR T-cells for this study. Both types are designed to find and destroy the plasma cells causing AL amyloidosis by targeting specific markers on their surface. This is a Phase 1 study, meaning it's one of the first times this treatment is being tested in humans. The main goals are to check if the treatment is safe and to see if it can help control AL amyloidosis in people who haven't responded to other treatments. It's hoped that this new therapy could offer a different way to fight the disease.

Key takeaways

This study evaluates a new CAR T-cell therapy for AL amyloidosis.
It targets patients whose disease has returned or not responded to previous treatments.
Your own immune cells are modified to fight the disease.
It's a Phase 1 study, focusing on safety and initial effectiveness.
Potential serious side effects include CRS and neurotoxicity, which are closely managed.

Who may be eligible?

This study is particularly for adults (aged 18 or over) who have been diagnosed with AL amyloidosis. Before considering this study, you must have tried at least one other treatment for your AL amyloidosis, and either that treatment didn't work as well as hoped, or your disease has unfortunately returned.

To make sure the treatment is as safe as possible for you, the study team will check your overall health. This includes making sure your organs like your kidneys and heart are working adequately, and that your blood counts (like white blood cells) are within a suitable range. You'll also need to be able to go through the study procedures and give your clear consent to take part.

There are also some reasons why you might not be able to join, such as having certain other specific health conditions like other types of amyloidosis, very low blood pressure, or being on kidney dialysis. The study team will review all your medical information carefully to see if this trial is right for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Have you been diagnosed with AL amyloidosis?
Has your AL amyloidosis returned or not responded well to at least one previous treatment?
Is your general health, including your organ function and blood counts, considered adequate by a doctor?
Do you not have any other specific types of amyloidosis or specific serious conditions that would exclude you?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the first step is to collect your T-cells, which are a type of white blood cell. This is done through a process called 'leukapheresis,' where blood is taken from a vein, processed to collect the T-cells, and then returned to your body. These collected T-cells are then sent to a lab to be specially modified into CAR T-cells.

Before you receive the CAR T-cells, you'll have a short course of chemotherapy, usually with two different drugs. This is to make space in your body for the new CAR T-cells to work effectively. After this, the modified CAR T-cells will be given back to you through a drip into a vein. You will then stay in the hospital for at least two weeks so the medical team can closely monitor you and manage any side effects. You will also have regular follow-up appointments and blood tests for an extended period to check how you are doing and how the treatment is working. The study is expected to run from February 2025 until January 2034.

Potential risks and benefits

It's important to understand both the potential benefits and risks. The main hope is that these new CAR T-cells could help to control your AL amyloidosis, especially if other treatments haven't worked well for you. However, as this is a new type of treatment, there's no guarantee it will work for everyone, and it might not have the desired effect. There are known side effects associated with each part of the process. Collecting your T-cells might cause some discomfort, bruising, or tingling. The chemotherapy drugs used before CAR T-cell infusion can cause side effects like infections, nausea, and in rare cases, bladder irritation. The CAR T-cells themselves can cause serious side effects like 'cytokine release syndrome' (CRS), which can lead to fever, low blood pressure, and could affect organs, and 'Immune effector cell-associated neurotoxicity syndrome' (ICANS), which affects the brain and can cause confusion or seizures. These side effects are closely monitored and managed by the hospital team, including in intensive care if necessary. You are in control and can decide to withdraw from the study at any time.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University College London Hospitals NHS Foundation Trust
City only
London, United Kingdom
Leeds Teaching Hospitals NHS Trust
City only
Leeds, United Kingdom

Common questions

What is AL amyloidosis?

AL amyloidosis is a rare condition where abnormal protein 'amyloid' builds up in organs, causing damage. It happens because certain immune cells (plasma cells) in your bone marrow produce faulty protein pieces.

What are CAR T-cells?

CAR T-cells are your own immune cells that have been specially changed in a lab. They are 're-programmed' to find and attack the abnormal cells that cause AL amyloidosis when put back into your body.

What is a Phase 1 trial?

A Phase 1 trial is an early stage of testing a new treatment in people. The main goals are to check if the treatment is safe, what dosage is best, and if it shows any signs of working.

What are the common side effects of CAR T-cell therapy?

The main potential side effects are 'cytokine release syndrome' (CRS) which can cause fever and low blood pressure, and 'neurotoxicity' (ICANS) which can affect brain function. These are closely monitored and treated.

Who is running this study?

This study is sponsored by University College London (UCL) and managed by the Cancer Research UK & UCL Cancer Trials Centre (UCL CTC). It's funded by Autolus, a company that develops T-cell therapies.

How to find out more

. ALARIC Trial Manager

ctc.alaric@ucl.ac.uk +44 203 1084173 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.