Investigational

CAR-T therapy

We’re looking to see if INVESTIGATIONAL CAR-T THERAPY could help reshape your immune system to target harmful autoimmune activity.¹

Find out about clinical trials for investigational CAR-T therapy in lupus, myositis, diffuse cutaneous systemic sclerosis, and vasculitis.

Participation in clinical trials is voluntary. You are free to withdraw from the trial at any time without any impact on your usual healthcare.

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Investigational CAR-T in autoimmune disease

What is investigational CAR-T therapy?

What to expect in an investigational CAR-T therapy clinical trial

Potential risks and benefits of investigational CAR-T therapy

Clinical trials in investigational CAR-T therapy

WHY CURRENT TREATMENTS MAY 
NOT BE ENOUGH

In some , B cells in the immune system that are meant to protect the body start producing molecules that attack healthy tissues instead.^1,2 People often spend years on treatments that need regular dosing to help control the B cells’ activity.¹

But some of the harmful B cells stay in the body even after treatment, which can contribute to some people not getting the relief they need.¹

Researchers are testing other treatments, including those that target B cells, so we can understand potential options for people who do not respond to available treatments.

Could a once only

INVESTIGATIONAL CAR-T INFUSTION

Provide long lasting disease control?

Our clinical trials are looking to find out.

Investigational CAR-T therapy is specific to each patient. It is designed to remove the B cells that drive autoimmune diseases. Our clinical trials are looking to see how the immune system responds to investigational CAR-T therapy and what effect it has on autoimmune diseases.

We are running four Phase 2 clinical trials for investigational CAR-T therapy called the AUTOGRAPH trials. These are designed to evaluate the safety and effectiveness of investigational CAR-T therapy in four autoimmune diseases:

AUTOGRAPH CLINICAL TRIALS:

In some of these trials, we will be researching the safety and effectiveness of investigational CAR-T therapy compared with commonly used treatments. So it is possible that you will not receive the investigational therapy if you participate.

WHAT IS INVESTIGATIONAL
  CAR-T THERAPY?

When our immune system is working properly, it protects us from pathogens and other harmful substances.

But in autoimmune diseases, the immune system mistakenly starts to attack the body’s own healthy cells and organs.^1</sup

Novartis’s clinical trials will evaluate whether, and for how long, the investigational CAR-T therapy can reduce this disease activity.¹

HOW IS INVESTIGATIONAL CAR-T THERAPY THOUGHT TO WORK?

An important part of our immune system is two types of white blood cells called B cells and T cells.

What does a B CELL do?

B cells produce antibodies that help our bodies attack germs that cause disease.

What does a T CELL do?

T cells are responsible for identifying and removing infected or abnormal cells.

Some T cells directly remove these infected or harmful cells, others help guide the immune system to deal with them.

How does investigational CAR-T therapy work?

Investigational CAR-T therapy is personalized to each patient. It genetically modifies a person’s own T cells to target the harmful B cells in the body.¹

A person’s blood is taken, and some of their T cells are collected. These T cells are given “chimeric antigen receptors” (or CARs), turning them into CAR-T cells.¹ These CAR-T cells are genetically modified to recognize and remove harmful B cells. The CAR-T cells are returned to the person’s body, where they target the B cells that are causing the autoimmune disease.¹ The body can then potentially replace the abnormal B cells with cells that work correctly.¹

These clinical trials will measure the safety of a once-only infusion of investigational CAR-T therapy and what impact it could have on the severity of your disease and quality of life.

WHAT TO EXPECT IN AN INVESTIGATIONAL CAR-T CLINICAL TRIAL

If you are eligible and choose to join an investigational CAR-T therapy trial, your trial team will clearly explain everything you need to know. Here’s a quick overview of the main steps involved in the trial, among others:

Step 1

Collecting your T cells

During a process called “leukapheresis,” your blood is passed through a machine that separates and collects some of your T cells. The rest of your blood is then returned to your body.

Step 2

Making the CAR-T cells

Your T cells are sent to a lab where they are genetically modified so they can target harmful B cells. The modified cells are called CAR-T cells.

Step 3

Preparing for your investigational CAR-T therapy

Next, your body needs to get ready for the investigational CAR-T cells. In a process called “lymphodepletion,” you’ll be given an infusion of medicine designed to reduce the number of white blood cells in your body so there is space for the new investigational CAR-T cells.

Step 4

Receiving your investigational CAR-T therapy

We’ll start by giving you some premedications to help reduce the risk of any side effects. You’ll then be given the investigational CAR-T cells through a one-time infusion.

Step 5

Monitoring after your investigational CAR-T therapy

You will need to stay in the hospital for at least 2 weeks so we can closely monitor your health and make sure you’re doing well. For the following 2 weeks, you’ll need to stay less than 2 hours’ travel away from the hospital or trial clinic.

Step 6

Long-term follow-up

When you’ve finished the trial, you’ll be asked if you want to join a long-term follow-up trial. If you join, we‘ll monitor your health to see the long-term effects of investigational CAR-T therapy.

POTENTIAL BENEFITS AND RISKS OF INVESTIGATIONAL CAR-T THERAPY

Potential benefits of investigational CAR-T therapy

Investigational CAR-T therapy is a once-only infusion that targets the cells that are responsible for autoimmune diseases.¹

We are investigating CAR-T therapy to see if it can continue to eliminate these harmful cells, long term. This may take away the need for lifelong medication.¹

With the potential for symptom relief without ongoing medication, investigational CAR-T therapy may give people with autoimmune diseases an improved quality of life.^1,3

As part of the clinical trial, you will receive frequent health assessments and monitoring. All trial-related care will be provided at no cost. 

What’s more, people who participate in clinical trials have the opportunity to help shape the future of care and grow our understanding of potential new treatments.

Investigational CAR-T therapy is being tested and is not yet FDA-approved, so there is no guarantee there will be a personal benefit.

Potential risks of investigational CAR-T therapy

Like with all medicines, treatments, and therapies, there may be some side effects from the investigational CAR-T therapy. The trial team will closely monitor you for any signs of side effects. This monitoring is important as it allows us to identify and treat side effects early. Some of the main side effects are listed below, but as this is an investigational product, we don’t know everything about it yet so there may be others.

Possible side effects

Fast activation of CAR-T cells can lead to an excess of molecules called cytokines, which can cause Cytokine Release Syndrome (CRS). This is a potentially serious complication of investigational CAR-T therapy and, in most cases, happens in the two weeks after a CAR-T infusion.¹ This is one of the reasons you will need to stay in hospital to be monitored for 2 weeks.

Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) is a potentially serious side effect that involves the brain. It can cause a person to feel disoriented, tired, and have difficulty speaking. ICANS can happen up to a month after receiving investigational CAR-T therapy.Fast activation of CAR-T cells can lead to an excess of molecules called cytokines, which can cause Cytokine Release Syndrome (CRS). This is a potentially serious complication of investigational CAR-T therapy and, in most cases, happens in the two weeks after a CAR-T infusion.¹ This is one of the reasons you will need to stay in hospital to be monitored for 2 weeks.

Investigational CAR-T therapy temporarily reduces the number of B cells. It may reduce both the harmful B cells, responsible for autoimmune diseases, and the correctly working B cells. This may mean your immune system is weakened, making you more susceptible to infections.¹

Other side effects may also occur. You will be provided with a detailed list of possible side effects if you choose to find out more about these clinical trials.

Safety in the AUTOGRAPH trials

These clinical trials are being carried out to find out if investigational CAR-T therapy is safe and effective, and you will be monitored throughout the duration of the trial. Your condition could improve, stay the same, or get worse after receiving it.

THE AUTOGRAPH CLINICAL TRIALS ARE LOOKING TO SEE IF INVESTIGATIONAL CAR-T THERAPY COULD BE A POSSIBLE TREATMENT FOR AUTOIMMUNE DISEASES

To find out more about the AUTOGRAPH CAR-T therapy clinical trials and whether you might be eligible, choose the condition that applies to you from the links below.

A member of the trial team will be able to explain the trial in simple terms and look at your full medical history to help you find out if the trial is right for you. By requesting more information, you won’t be committing to join a trial.

AUTOGRAPH CLINICAL TRIALS:

SUPPORT FOR CAREGIVERS

Caring for a person with lupus is a vital role, and important to the success of a person’s treatment.

As part of the clinical trial, the person receiving the investigational CAR-T therapy will need to remain in hospital for 2 weeks after the infusion. They will then need to stay within 2 hours’ travel from the hospital for a further 2 weeks. It’s important that you’re aware of this as they may need your support during this time.

These are the key ways that caregivers can help:

Understanding

Help your loved one understand what will happen during the clinical trial and raise any questions with the trial team.

Monitoring

Watch for any changes in how they feel and share these with the healthcare team.

Emotional support

Being present, listening, and helping them manage stress can make the process easier.

Transport

You may need to help with getting to appointments and keeping track of schedules.

Remember, support is always available if you need it. Contact the trial team to discuss any help you may need.

COMMON QUESTIONS
ABOUT THE CLINICAL TRIALS

FAQs

What is a clinical trial?

Clinical trials (or clinical studies) are more than just research. They are an essential part of developing new medicines and thousands of people around the world take part in them every year. Everyone involved in a clinical trial is helping shape the future of care.

Clinical trials are a type of medical research that volunteers take part in to help us find out important information about a medicine, device, or procedure, such as:

- Does it work?
- What are the risks or side effects?
- Is it better than existing treatment options?

Clinical trials can offer patients access to investigational therapies with care and monitoring for the duration of the clinical trial.

More information about clinical trials is available in this booklet [link].

What is the investigational CAR-T therapy that is being studied?

Investigational CAR‑T therapy is a personalized therapy made from each patient’s own immune cells. It modifies a person’s immune system to target harmful B cells that contribute to autoimmune diseases.

These clinical trials are looking at how effective investigational CAR‑T therapy is at helping people with autoimmune diseases who have been relying on the long-term use of other medications. Investigational CAR-T therapy is being tested and is not yet FDA-approved, so there is no guarantee there will be a personal benefit.

What are the trial objectives?

The AUTOGRAPH trials are Phase 2 trials to learn about Novartis’s investigational CAR-T therapy.

We are investigating its safety and effectiveness to see if it can help improve the lives of people with severe or refractory autoimmune diseases. Throughout the trial your autoimmune disease will be monitored to see if it has improved after treatment. Your overall health will also be monitored for any signs of side effects.

There are four separate trials, each looking at Novartis’s investigational CAR-T therapy in autoimmune diseases which are known to involve B cells. These are lupus (SLE / LN), myositis (IIM), diffuse cutaneous systemic sclerosis (dcSSc), and vasculitis (AAV).

What to expect if you participate in an AUTOGRAPH trial

Overall, the Novartis investigational CAR-T trials (known as the AUTOGRAPH clinical trials) will last for up to 6 years, depending on which trial you enroll in. The trials have several stages. If you are receiving investigational CAR-T therapy the stages will be:

- Screening: Assessments are carried out to make sure that you and the trial are a good fit
- Pre-treatment: We’ll collect T cells from your blood (see What to expect with investigational CAR-T therapy [link]) so that we can make your investigational treatment. You’ll also be given medicines to prepare your body for the investigational CAR-T therapy
- Treatment: You’ll be given your investigational treatment in a one-time infusion into the vein. After that you’ll need to stay in hospital for at least 2 weeks so the trial team can closely monitor you. After that you will need to stay within 2 hours’ travel of the hospital for a further 2 weeks
- Follow-up: After you’ve been discharged from hospital, you’ll need to come to the trial clinic for additional visits so that we can continue to monitor your health

Long-term follow-up trial: Once you have completed the trial, you may be asked to join a long-term follow-up trial.

Who can take part in the trials?

We are looking for people to take part in clinical trials for Novartis’s investigational CAR-T cell therapy. To be included, we need people who, among other criteria:

Are between 18 and up to 75 years old
Have severe autoimmune disease, either lupus (SLE / LN), myositis (IIM), diffuse cutaneous systemic sclerosis (dcSSc), or vasculitis (AAV), and have not responded well to previous treatments
Have adequate organ function (how well your organs are working)
Do not have other medical conditions that would prevent their ability to tolerate lymphodepletion (lowering specific white blood cells temporarily) and investigational CAR-T therapy

For more information about the trial and other eligibility criteria, see the links below. If you aren’t sure if this applies to you, please contact your doctor or the trial team for more information. The team will look at your full medical history to help you find out if the trial is right for you. By requesting more information, you won’t be committing to join a trial.

AUTOGRAPH – Lupus

AUTOGRAPH – Vasculitis

AUTOGRAPH – Diffuse cutaneous systemic sclerosis

AUTOGRAPH – Myositis

What support is available during the trial?

Clinical sites may be able to support you financially and with travel so that we can ensure you have the easiest experience possible. They may be able to support with travel coordination, compensation for trial visits, and visit-associated expenses.

Reimbursement policies vary by site and are subject to ethics committee approval. Speak to the trial team for more detailed information on support that may be available to you.

Glossary

Antibodies Proteins made by your immune system to fight infections.
AUTOGRAPH The name given to the clinical trials for Novartis’s investigational CAR-T therapy.
Autoimmune diseases A group of diseases in which a person’s immune system attacks their own body.
B cell A type of white blood cell that helps protect your body by making antibodies. In some diseases, B cells can become overactive or damaged, which is why treatments like CAR-T cell therapy may target them.
CAR (Chimeric Antigen Receptor) A special protein added to your T cells in the lab so they can recognize and attach to certain cells in your body. This change helps your T cells to find and destroy the diseased cells once they are infused back into you.
CAR-T cell Your own immune cells (T cells) that have been modified in a lab to recognize and target harmful B cells.
Cytokines Natural chemical messengers made by immune cells that help them communicate.

Effectiveness/efficacy How well a therapy, treatment, or medicine works.
Infusion When medicine or CAR-T cells are given slowly into a vein through an intravenous (IV) or central line.

Leukapheresis A procedure where some of your white blood cells (T cells) are collected from your blood using a special machine. The rest of your blood is returned to your body. These cells are later genetically modified in the lab to make CAR-T cells.
Lymphodepletion A short course of medicine given before your investigational CAR-T therapy infusion to make space in your immune system and help the new investigational CAR-T cells work better.
Molecule A biological building block that has a specific function in the body.
Monitoring Regular checks, examinations, and visits done by the trial team to follow your health and safety during and after treatment.
Pathogen An organism, e.g., bacteria, virus, or fungus, that can cause an infection.
Phase 2 (clinical trial phase) The stage of a clinical trial where a treatment is given to a larger group of people (often 50–200) to investigate how well it works and to learn more about its safety.
Refractory Describes a disease which is not responding well to treatment.
Safety Whether a therapy, treatment, or medicine has side effects/adverse events, what the side effects are, and how serious they are.
Severe Describes a disease that is very bad or intense, and which has a major impact on daily life.
Side effect (adverse event) An unwanted or unexpected reaction to a medicine or treatment, such as fever, nausea, or tiredness. Some are mild and temporary, while others may need medical care.
Side effect (adverse event) An unwanted or unexpected reaction to a medicine or treatment, such as fever, nausea, or tiredness. Some are mild and temporary, while others may need medical care.

Trial team The group of doctors, nurses, coordinators, pharmacists, and other professionals who manage the trial and patient care.
T cell A type of white blood cell that helps your body fight infections and diseases. In investigational CAR-T therapy, your own T cells are collected and changed in a lab so they can better recognize and attack harmful cells.
Tissue A group of cells that combine together to form a bigger structure e.g., an organ, muscle, or tendon.

Novartis Clinical Trials

For more information about Novartis clinical trials for patients and caregivers, please visit our Novartis website.

References

Rangel-Peláez C, et al. Front Immunol. 2024;15:1502712.
Merino-Vico A, et al. Eur J Immunol. 2023;53:e2149675.
Merino-Vico A, et al. Int J Mol Sci. 2021;23:387.