The AUTOGRAPH-SSc

trial

Investigational CAR-T in autoimmune disease

We’re looking to see if investigational CAR-T therapy could help reshape your immune system to target harmful autoimmune activity in diffuse cutaneous systemic sclerosis (dcSSc).1

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Investigational CAR-T in autoimmune disease
New treatments are needed for dcSSc

About the AUTOGRAPH-SSc trial

Are you eligible?

What to expect

What will you need to do?

Support for caregivers

What’s next?

Common questions

NEW TREATMENTS

ARE NEEDED FOR dcSSc

Currently available treatments for dcSSc only focus on the symptoms or the problems that may occur in patients’ organs as a result of their dcSSc, meaning they don’t target the root cause of the disease.

Medicines that reduce immune activity are sometimes used, but some people experience side effects that they’re unable to tolerate.

Others may not respond as hoped.

Or they respond well but then find that their medication no longer gives them the same benefits.

Other treatments that remove B cells are also used , but these don’t eliminate all the harmful B cells. So, there is a significant need for targeted and more effective treatment options for people with dcSSc.

CAR-T cell
This clinical trial is researching investigational CAR-T × CAR-T cell Your own immune cells (T cells) that have been modified in a lab to recognize and target harmful B cells. therapy in people with dcSSc that have not responded to available treatments.

The trial aims to understand safety and the impact it could have on the severity of your disease and quality of life.

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

How diffuse cutaneous systemic sclerosis works

In people with systemic sclerosis (specifically diffuse cutaneous systemic sclerosis), the immune system mistakenly attacks connective tissue, leading to fibrosis (scarring) that affects the skin and internal organs, particularly the lungs.2

B cells have been found to be a key factor in the immune processes that cause dcSSc, making them an important target for treatment. 3

B Cell
Some treatments aim to lower B-cell × 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 therapy may target them. activity to improve dcSSc symptoms, requiring years of regular dosing. Other treatments aim to reduce the number of harmful B cells in the body. Unfortunately, many patients find they get little benefit from these medicines because they don’t reduce the B cells enough.1

Investigational CAR-T cell therapy in dcSSc

Existing medications are designed to reduce and control B-cell activity, often with multiple, repeated doses. Whereas investigational CAR-T cell therapy aims to reduce the B cells that drive dcSSc, with a one-time infusion × Infusion When medicine or CAR-T cells are given slowly into a vein through an intravenous (IV) or central line. .

Personalized to each patient, it uses your own immune cells – T cells – that are collected from your blood and genetically modified in a lab so they can find and remove harmful B cells.

The modified cells are then returned to your body in a one-time infusion. This trial is testing the safety and effectiveness of investigational CAR-T therapy in people with dcSSc.1

Investigational CAR-T therapy is being studied as a possible option for people with severe × Severe Describes a disease that is very bad or intense, and which has a major impact on daily life. dcSSc that have not responded to available treatments.

Learn more about CAR-T cell therapy

The clinical trial

The AUTOGRAPH × AUTOGRAPH The name given to the clinical trials for Novartis’s investigational CAR-T therapy. -SSc clinical trial is studying investigational CAR-T cell therapy for adults with severe diffuse cutaneous systemic sclerosis.

The trial aims to understand how safe this investigational treatment is and whether it can help control disease activity when existing treatments have not worked well in people with diffuse cutaneous systemic sclerosis (or dcSSc).

CAR-T cell

ABOUT THE AUTOGRAPH-SSc TRIAL

The AUTOGRAPH-SSc clinical trial is assessing the safety × Safety Whether a therapy, treatment, or medicine has side effects/adverse events, what the side effects are, and how serious they are. and effectiveness × Effectiveness/efficacy How well a therapy, treatment, or medicine works. of an investigational CAR-T cell therapy in people with dcSSc.

The trial is designed to learn whether this investigational treatment may help people whose disease has continued to progress despite available therapies.

Keep reading to find out more about the purpose of the trial. We’ll talk about the details of how the trial works, what treatment you may receive, and what taking part involves.

What is the aim of this clinical trial?

The aim of the AUTOGRAPH-SSc clinical trial is to better understand how safe investigational CAR-T cell therapy is and how well it works compared with rituximab in people with dcSSc...

The aim of the AUTOGRAPH-SSc clinical trial is to better understand how safe investigational CAR-T cell therapy is and how well it works compared with rituximab in people with dcSSc.

Researchers are looking to see if dcSSc patients who have been relying on the long-term use of other medications may be able to experience sustained improvement of their disease and quality of life with a once-only CAR-T infusion × Infusion When medicine or CAR-T cells are given slowly into a vein through an intravenous (IV) or central line. .

Information from this trial may help improve future treatment options for people with dcSSc. This section provides a high-level overview.

More details about the trial process, treatment options, and what taking part involves are explained below.

What medications could I receive on this trial?

This is a Phase 2 × 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. trial, meaning we are investigating the safety and effectiveness of investigational CAR-T × CAR-T cell Your own immune cells (T cells) that have been modified in a lab to recognize and target harmful B cells. cell therapy compared with commonly used treatments. Not everyone in the trial will receive investigational CAR-T therapy; some people will receive existing treatments so we can compare any changes fairly...

This is a Phase 2 × 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. trial, meaning we are investigating the safety and effectiveness of investigational CAR-T × CAR-T cell Your own immune cells (T cells) that have been modified in a lab to recognize and target harmful B cells. cell therapy compared with commonly used treatments. Not everyone in the trial will receive investigational CAR-T therapy; some people will receive existing treatments so we can compare any changes fairly.

If you choose to take part in this trial, you may receive either:

  • The investigational CAR-T cell therapy being studied, or
  • Rituximab, or a standard of care available option. However, in some cases, if you don’t respond well to these, you may have the option to switch to investigational CAR-T therapy.

The trial team will explain the available options and how treatment decisions are made before you decide whether to take part.

What support is available during the trial?

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

Clinical sites may be able to support you financially and with travel so that we can ensure patients have the easiest experience possible. They may also 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 approval. Speak to the trial team for more detailed information on support that may be available to you.

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 participate in them every year...

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 participate 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 human volunteers take part in, and help us find out important information about a medicine, device, test, or procedure, such as:

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

Clinical trials can offer patients access to investigational therapies with care and monitoring × Monitoring Regular checks, examinations, and visits done by the trial team to follow your health and safety during and after treatment. for the duration of the clinical trial.

More information about clinical trials is available in this clinical trials booklet.

ARE YOU ELIGIBLE?

This trial is for adults with severe, progressive diffuse cutaneous systemic sclerosis.

Whether you can take part depends on several medical factors and should always be discussed with your doctor.

You may be able to take part if you:

  • Are an adult (between 18 and 70)
  • Have been diagnosed with systemic sclerosis, specifically diffuse cutaneous systemic sclerosis within 7 years before screening
  • Have severe, progressive disease that includes at least one of the following dcSSc-related conditions:
    • dcSSc-associated lung disease
    • Severe, progressive dcSSc skin disease
    • Significant dcSSc-associated heart problems
  • Have non-Raynaud symptoms, e.g. puffy hands/fingers, skin tightening, joint pain, digital ulcers, and severe fatigue
  • Have not responded well to previous treatments
  • Are able to attend study visits and follow study requirements
  • Have received all recommended vaccinations for immunocompromised patients

You may not be able to take part if you:

  • Have another rheumatic disease other than dcSSc, including limited cutaneous systemic sclerosis (lcSSc) or sine scleroderma
  • Have any condition that means a complete washout of medications is not possible
  • Have a hypersensitivity to rituximab or any of the ingredients in Novartis’s CAR-T therapy
  • Have other health issues that would prevent you from receiving the study treatments, including but not limited to:
    • Pre-existing pulmonary hypertension
    • Kidney problems
    • Uncontrolled stage 2 hypertension
  • Have been vaccinated with live attenuated vaccines within 6 weeks before starting the study
The trial team × Trial team The group of doctors, nurses, coordinators, pharmacists, and other professionals who manage the trial and patient care. will review your full medical history during screening to help decide if this study may be right for you.
For more detailed eligibility information, see clinicaltrials.gov and talk to your healthcare professional.

WHAT TO EXPECT

If you are going to receive the investigational CAR-T cell therapy, the timings will follow the timeline below. If you receive “ standard of care × Standard of care treatments The usual treatments that are commonly used by doctors to manage a condition. These treatments are based on current medical guidelines and are considered appropriate care outside of a clinical trial. ,” your healthcare professional will discuss the timeline with you separately.

Screening period (up to 6 weeks)

Assessments are carried out to make sure that you and the trial are a good fit

Treatment period (2 weeks)

Receiving your investigational CAR-T therapy

You’ll be given your investigational treatment in a one-time infusion into the vein

Monitoring after your investigational CAR-T therapy

After the infusion, you’ll need to stay in hospital for at least 2 weeks so the trial team can closely monitor × Monitoring Regular checks, examinations, and visits done by the trial team to follow your health and safety during and after treatment. you. After that you will need to stay within 2 hours’ travel of the hospital for a further 2 weeks

Long-term follow-up trial (up to 15 years from investigational CAR-T cell therapy)

Once you have completed the trial, you may be asked to join a long-term follow-up trial for up to a combined total of 15 years from receiving investigational CAR-T therapy, to understand more about the ongoing effects of the investigational CAR-T cell therapy
Participation in the follow-up trial is voluntary. You don’t have to be involved in the long-term follow-up trial, even if you participated in the main trial

Pre-treatment period (4 weeks)

Collecting your T cells

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

Making the CAR-T cells

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

Preparing for your investigational CAR-T therapy

Next, your body needs to get ready for the investigational CAR-T × CAR-T cell Your own immune cells (T cells) that have been modified in a lab to recognize and target harmful B cells. cells. In a process called “ lymphodepletion × 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. ,” you’ll be given an infusion × Infusion When medicine or CAR-T cells are given slowly into a vein through an intravenous (IV) or central line. 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

You’ll be given medicines to control your systemic sclerosis

Follow-up (up to 5 years)

For up to 8 weeks after CAR-T treatment, you will need to come into the hospital at least once a week. Visits can take up to 4 hours
After this initial period, visits become less frequent

Screening period (up to 6 weeks)

Assessments are carried out to make sure that you and the trial are a good fit

Pre-treatment period (4 weeks)

Collecting your T cells

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

Making the CAR-T cells

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

Preparing for your investigational CAR-T therapy

Next, your body needs to get ready for the investigational CAR-T × CAR-T cell Your own immune cells (T cells) that have been modified in a lab to recognize and target harmful B cells. cells. In a process called “ lymphodepletion × 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. ,” you’ll be given an infusion × Infusion When medicine or CAR-T cells are given slowly into a vein through an intravenous (IV) or central line. 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

You’ll be given medicines to control your systemic sclerosis

Treatment period (2 weeks)

Receiving your investigational CAR-T therapy

You’ll be given your investigational treatment in a one-time infusion into the vein

Monitoring after your investigational CAR-T therapy

After the infusion, you’ll need to stay in hospital for at least 2 weeks so the trial team can closely monitor × Monitoring Regular checks, examinations, and visits done by the trial team to follow your health and safety during and after treatment. you. After that you will need to stay within 2 hours’ travel of the hospital for a further 2 weeks

Follow-up (up to 5 years)

For up to 8 weeks after CAR-T treatment, you will need to come into the hospital at least once a week. Visits can take up to 4 hours

After this initial period, visits become less frequent

Long-term follow-up trial (up to 15 years from investigational CAR-T cell therapy)

Once you have completed the trial, you may be asked to join a long-term follow-up trial for up to a combined total of 15 years from receiving investigational CAR-T therapy, to understand more about the ongoing effects of the investigational CAR-T cell therapy

Participation in the follow-up trial is voluntary. You don’t have to be involved in the long-term follow-up trial, even if you participated in the main trial

WHAT WILL YOU NEED TO DO?

Taking part in the AUTOGRAPH × AUTOGRAPH The name given to the clinical trials for Novartis’s investigational CAR-T therapy. -SSc clinical trial is completely voluntary. You can choose not to take part or to leave the trial at any time.

If you do decide to enroll, participation involves a long-term commitment. This includes attending trial visits, following personalized treatment plans, and making temporary adjustments to daily routines.

The sections below explain these commitments so you can decide whether this trial may be right for you.

Attending trial visits

The trial stages include a pre-treatment period, the treatment itself, and regular follow-up visits. These follow-up visits are important so the trial team can closely monitor × Monitoring Regular checks, examinations, and visits done by the trial team to follow your health and safety during and after treatment. your health and learn more about how the treatment is working.
The trial team × Trial team The group of doctors, nurses, coordinators, pharmacists, and other professionals who manage the trial and patient care. will explain the visit schedule in detail and help you plan ahead.

Treatment plans

Details of this will need to be discussed with your doctor and the trial team. There are some treatments that cannot be taken whilst on the trial.

You may need to stop taking your current treatment before your T cells are collected. This is called a washout period. The trial team will support you to make sure this is done as safely as possible.

If you are receiving a class of medicine known as glucocorticoids (GCs), such as prednisone or dexamethasone, you may need to stop or reduce your dose.

After treatment

You will need to stay in hospital for at least 2 weeks after the investigational CAR-T × CAR-T cell Your own immune cells (T cells) that have been modified in a lab to recognize and target harmful B cells. therapy is given. After that, you will need to stay within 2 hours’ travel from the hospital for the next 2 weeks.
This may have an impact on your ability to go to work. It may help to talk to your employer about flexible working or medical leave. The trial team can help if you need support with these conversations.

Things to avoid

You will need to avoid driving or operating heavy machinery for 8 weeks after receiving investigational CAR-T therapy.

It is not known whether the trial treatment(s) may harm an unborn or nursing baby. If you are pregnant, trying to become pregnant, or breastfeeding, you cannot be in the trial.

There are requirements for contraception for both female and male participants from the time they join and for at least 2 years after receiving the investigational CAR-T therapy.

Cyclophosphamide (used as one of the pre-treatment medications for lymphodepletion) can affect male and female fertility. Your trial team will discuss your potential options for fertility preservations, including risks and outcomes.

You should not donate blood, organs, tissues × Tissue A group of cells that combine together to form a bigger structure e.g., an organ, muscle, or tendon , sperm, eggs, or other cells after receiving investigational CAR-T therapy.

SUPPORT FOR CAREGIVERS

Caring for a person with dcSSc 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 × Trial team The group of doctors, nurses, coordinators, pharmacists, and other professionals who manage the trial and patient care. to discuss any help you may need.

WHAT’S NEXT?

To find out more about the AUTOGRAPH-SSc CAR-T therapy clinical trial and whether you might be eligible, click the link below.

Check eligibility
A member of the trial team × Trial team The group of doctors, nurses, coordinators, pharmacists, and other professionals who manage the trial and patient care. 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. You won’t be committing to join the trial.

Informed consent

Before anything else happens, you’ll meet with the trial doctor (also called the principal investigator). They will explain the purpose of the trial and who may be able to take part.

The trial team will share the informed consent form and other helpful materials, including short videos. These explain what taking part involves, the possible risks and benefits, and the time commitment.

Informed consent means you take time to learn about the trial, ask questions, and decide whether you want to take part. If you choose to continue, you’ll sign the consent form to confirm that you’re joining voluntarily.

Screening

After giving informed consent, you’ll go through a screening process. This helps the trial team check whether the trial is a good and safe fit for you.

Screening may include a review of your medical history, physical exams, and medical assessments. The goal is to confirm that you meet the trial’s requirements and do not have any conditions that would make participation unsafe.

The trial team will explain each step and answer any questions you may have.

Enrollment

If screening shows that you meet all trial criteria, you may be enrolled in the trial. The trial site will confirm your eligibility and guide you through the next steps.

Being enrolled means you can move forward in the trial, but participation remains voluntary throughout. You can choose to stop taking part at any time.

COMMON QUESTIONS

Common questions about the clinical trial

After you contact a trial site, the trial team 
×
Trial team
The group of doctors, nurses, coordinators, pharmacists, and other professionals who manage the trial and patient care. will guide you through a screening process to see if the trial is a good and safe fit for you.

This includes reviewing the informed consent form, your medical history, and doing routine exams and health assessments, such as blood and urine tests, vital signs, and heart checks.

If screening shows that you don’t qualify, the trial team will explain why, answer your questions, and talk with you about other care or research options.

Find out more about eligibility or contact a trial team here. By requesting more information, you won’t be committing to join a trial.

Check eligibility
No, you can contact the trial team × Trial team The group of doctors, nurses, coordinators, pharmacists, and other professionals who manage the trial and patient care. to find out more information without committing to taking part in a clinical trial.

If you are interested, the trial team will discuss the risks and benefits with you before you make your decision. As a volunteer, you can choose to leave at any time without any penalty or loss of medical benefits.
No. Participants will receive investigational CAR-T × CAR-T cell Your own immune cells (T cells) that have been modified in a lab to recognize and target harmful B cells. therapy and trial-related medical care and assessments at no cost.

You will not have to pay for any trial treatment(s) or any trial-related assessments or procedures while you are participating in the trial. However, the Sponsor will not pay for the costs of the usual care you need to maintain your health.

We understand that taking part may lead to other costs, such as time off work. Support from the trial sponsor may be available to help with this.

Yes. This trial is unblinded, which means both you and your trial doctor will know which treatment you are receiving. Some trial staff who assess your disease during the trial will not know which treatment you’re on. This helps keep the trial results as fair and accurate as possible.

Details of this will need to be discussed with your doctor and the trial team × Trial team The group of doctors, nurses, coordinators, pharmacists, and other professionals who manage the trial and patient care. . There are some treatments that cannot be taken whilst on the trial.

You may need to stop taking your current treatment before your T cells × 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. are collected. This is called a washout period. The trial team will support you to make sure this is done as safely as possible.

If you are receiving a class of medicine known as glucocorticoids (GCs), such as prednisone or dexamethasone, you may need to reduce your dose.
Maybe. You will need to stay in hospital for at least 2 weeks after the investigational CAR-T × CAR-T cell Your own immune cells (T cells) that have been modified in a lab to recognize and target harmful B cells. therapy is given. After that, you will need to stay within 2 hours’ travel from the hospital for the next 2 weeks. This may have an impact on your ability to work. It may help to talk to your employer about flexible working or medical leave. The trial team × Trial team The group of doctors, nurses, coordinators, pharmacists, and other professionals who manage the trial and patient care. can help if you need support with these conversations.
At your trial visits, the team will check how you’re doing and monitor × Monitoring Regular checks, examinations, and visits done by the trial team to follow your health and safety during and after treatment. your health. This may include talking about how you’ve been feeling, physical exams, heart checks (such as an ECG), blood and urine tests, and questionnaires about your disease and overall health. Some visits may also include imaging scans, such as a computed tomography (CT) scan.
Most trial procedures are simple and cause little discomfort. Blood tests may briefly sting or cause mild bruising.

Some people may experience side effects × 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. from procedures required before treatment. Leukapheresis × 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. is generally well tolerated but can cause temporary symptoms such as tingling in the lips or fingers, dizziness, or bruising where the needles are placed. Medicines given before treatment may also cause side effects, including nausea, fatigue, or increased risk of infection.

The trial team will monitor × Monitoring Regular checks, examinations, and visits done by the trial team to follow your health and safety during and after treatment. you closely and explain what to expect at each step.

In most cases, yes. Visitor policies depend on the hospital, so rules may vary. Because your immune system may be weaker, visitors might need to follow safety measures such as wearing masks or washing their hands carefully. Your trial team can explain the visiting hours and any guidelines you or your visitors will need to follow.

No. If you are not receiving the investigational CAR-T therapy, you will continue with safety and effectiveness follow-up visits while receiving the comparison treatment. There is no planned hospital stay for people in this part of the trial.

Long-term follow-up is a separate and optional program for participants who receive the investigational CAR-T therapy. It helps the trial team × Trial team The group of doctors, nurses, coordinators, pharmacists, and other professionals who manage the trial and patient care. monitor health and safety over time.

This follow-up may last for up to 15 years from receiving investigational CAR-T × CAR-T cell Your own immune cells (T cells) that have been modified in a lab to recognize and target harmful B cells. cell therapy and usually includes check-ins every six months. Visits may be in person or by phone. In-person visits may include blood and urine tests and questionnaires to help track overall health and monitor × Monitoring Regular checks, examinations, and visits done by the trial team to follow your health and safety during and after treatment. the CAR-T cells in the body.

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.
  • Standard of care treatments The usual treatments that are commonly used by doctors to manage a condition. These treatments are based on current medical guidelines and are considered appropriate care outside of a clinical trial.
  • 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.

Novartis Clinical Trials

Further information on autoimmune diseases

To learn more about autoimmune diseases, please visit these independent patient advocacy organizations:

Lupus

Lupus and Allied Diseases Association
Lupus Foundation of America
Lupus Research Alliance

Myositis

The Myositis Association
Myositis Support & Understanding

Diffuse cutaneous systemic sclerosis

National Scleroderma Foundation
Scleroderma Research Foundation

Vasculitis

Vasculitis Foundation
Vasculitis International

References

  1. Rangel-Peláez C, et al. Front Immunol. 2024;15:1502712.
  2. Herrick AL, et al. Nat Rev Rheumatol. 2022;18:276–285.
  3. Beesley CF, et al. Front Immunol. 2023;13:999008.

Data We Collect & Why:

  • Reply to your requests, inquiries and comments;
  • Respond to circumstances permitted or required by law, including defending and bringing legal actions; and
  • If applicable, only on clinical trial recruitments web sites, to determine whether you may be eligible to participate in Sponsor’s Study. During the recruitment period for the Study, we may provide your screening information and contact information to one of our Sponsor’s investigator sites nearest to you.

To control data you have already submitted, make a data request here.

Data you submit is handled according to our Privacy Policy and web site user agreements.

We Do Not Sell Your Data to 3rd Parties

We do not sell data to third parties. Data you submit is handled according to our Privacy Policy and web site use agreements.

To control data you have already submitted, or to log a do not sell data request, make a data request here.