RecruitingPHASE1, PHASE2INTERVENTIONAL

A Study to Investigate the Safety, Tolerability, and Efficacy of AZD0120 in Adults With Refractory SLE

This research study is looking at a new type of treatment called AZD0120 for adults with a severe form of lupus, specifically Systemic Lupus Erythematosus (SLE), that hasn't improved with other standard treatments. Lupus is a condition where the body's immune system mistakenly attacks its own tissues. The study has two main parts: first, to check if AZD0120 is safe and well-tolerated, and then to see how effective it is in reducing lupus symptoms. AZD0120 is a special kind of 'CAR T-cell therapy', which means it uses a person's own immune cells that are specially modified in the lab to target and fight certain cells linked to lupus. The study also uses standard medicines like Cyclophosphamide and Fludarabine alongside the new treatment.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

AstraZeneca

Enrolment target

150

Start

21 Apr 2025

Estimated completion

01 May 2029

Lupus Erythematosus, Systemic

What is this study about?

This study is about a new treatment called AZD0120 for a condition called Systemic Lupus Erythematosus (SLE). SLE, often just called lupus, is a long-term condition where your body's immune system, which normally fights off infections, starts to attack its own healthy tissues and organs. This can cause a wide range of symptoms like joint pain, tiredness, skin rashes, and problems with kidneys or other organs. The treatment being tested, AZD0120, is for people whose lupus is 'refractory', meaning it hasn't responded well to other standard medications.

AZD0120 is a very advanced type of treatment called 'CAR T-cell therapy'. It involves taking some of your own immune cells, called T-cells, from your blood. These cells are then sent to a lab where they are specially changed to help them recognise and fight cells that are believed to play a role in lupus. Once these modified cells are grown in large numbers, they are put back into your body through a drip. The idea is that these new, specially trained cells will help to rebalance your immune system and reduce the lupus activity.

Before you receive the AZD0120 treatment, you would also be given some common chemotherapy drugs, Cyclophosphamide and Fludarabine. These are used to prepare your body for the CAR T-cells. The study is split into two phases: the first part focuses on finding the safest and best-tolerated dose of AZD0120. Once that's established, the second part will look more closely at how well this treatment works to improve lupus symptoms. This research is important because it explores new ways to help people with severe lupus who haven't found relief with existing medicines.

Key takeaways

This study is testing a new cell therapy (AZD0120) for severe lupus that hasn't improved with other treatments.
It aims to check the safety, tolerability, and effectiveness of this new approach.
The treatment involves using your own specially modified immune cells.
Participants will also receive standard chemotherapy drugs as part of the process.
It's for adults aged 18-70 with specific types of active lupus.
Participation involves multiple hospital visits and long-term follow-up.

Who may be eligible?

To be considered for this study, you would need to be an adult between 18 and 70 years old. You must have a clear diagnosis of Systemic Lupus Erythematosus (lupus) that has been confirmed by specific medical tests, and your lupus must be active and severe enough, and it needs to have not responded well to at least two other types of standard lupus medication, including one 'biological agent'. Your doctors would also need to see that you have specific lupus-related problems affecting an organ, such as your joints, skin, heart, or kidneys.

There are also some important health checks you'd need to pass. For example, your liver, kidneys, lungs, and heart would need to be working well. You also couldn't have certain other health problems, such as some types of cancer that are growing, or active infections. If you've had certain other advanced cell therapies or bone marrow transplants in the past, or treatments targeting specific immune markers (CD19 and/or BCMA), you wouldn't be able to join this study.

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 between 18 and 70 years old?
Do you have a diagnosis of Systemic Lupus Erythematosus (lupus)?
Has your lupus not responded well to at least two other treatments, including a 'biological' medicine?
Do you have active lupus affecting certain organs (e.g., joints, skin, kidneys)?
Are your liver, kidneys, lungs, and heart generally healthy?
Have you NOT had certain advanced cell therapies or bone marrow transplants before?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, it would involve several visits to the study clinic or hospital over a period of time. Initially, there will be screening appointments to check if you meet all the requirements. If you do, you would have blood tests to collect your T-cells, followed by a waiting period while your cells are modified in the lab. Before receiving the AZD0120, you would have an infusion of two common chemotherapy drugs (Cyclophosphamide and Fludarabine) for a few days to prepare your body. Then, the modified AZD0120 cells would be given back to you through a drip. After the treatment, there would be many follow-up visits, which could involve blood tests, physical exams, and checking your lupus symptoms. The study team would closely monitor your health for a significant period to make sure the treatment is safe and to see how well it's working. The exact number and timing of visits would be explained in detail, and the total duration of your participation, including follow-up, could be several years.

Potential risks and benefits

Like all medical studies, there are potential benefits and risks. The potential benefit is that this new treatment, AZD0120, could help improve your lupus if other treatments haven't worked. However, there's no guarantee it will work for everyone, and it's an investigational treatment, meaning we don't fully know its effects yet. Potential risks include side effects from the chemotherapy drugs used and the AZD0120 treatment itself, which could range from mild to serious, as CAR T-cell therapies can sometimes affect the immune system in unexpected ways. The study team will explain all known risks to you. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (20)

Research Site
Verified postcode
San Francisco, United States· Not yet recruiting
Research Site
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Stanford, United States· Not yet recruiting
Research Site
Verified postcode
Tampa, United States· Suspended
Research Site
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Atlanta, United States· Not yet recruiting
Research Site
Verified postcode
Chicago, United States· Not yet recruiting
Research Site
Verified postcode
Baltimore, United States· Not yet recruiting
Research Site
Verified postcode
Bethesda, United States· Withdrawn
Research Site
Verified postcode
Boston, United States· Recruiting
Research Site
Verified postcode
St Louis, United States· Recruiting
Research Site
Verified postcode
New York, United States· Recruiting
Research Site
Verified postcode
Syracuse, United States· Recruiting
Research Site
Verified postcode
Chapel Hill, United States· Recruiting

Common questions

What is Systemic Lupus Erythematosus (SLE)?

SLE, or lupus, is a condition where your body's immune system mistakenly attacks its own healthy tissues and organs, causing inflammation and damage throughout the body.

What does 'refractory' lupus mean?

Refractory lupus means that your lupus hasn't responded well to standard medications, and your symptoms continue to be active and problematic despite treatment.

What is AZD0120?

AZD0120 is a new, experimental 'CAR T-cell therapy'. It involves modifying your own immune cells in a lab to help them fight against certain cells linked to lupus activity.

Will I have to stay in hospital for this study?

Yes, you would likely need to stay in the hospital for some parts of the treatment, especially around the time you receive the chemotherapy and the AZD0120 cells, for close monitoring.

Is this a new treatment or one that's already available?

AZD0120 is an investigational new treatment, meaning it's still being tested in clinical trials and is not yet available for general use outside of research studies.

How to find out more

AstraZeneca Clinical Study Information Center

information.center@astrazeneca.com 1-877-240-9479 Official trial record

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