Not yet recruitingRare disease

A Study Testing Anti-CD19 CAR T-Cell Therapy in Patients with Systemic Sclerosis Who Did Not Respond to Immunosuppressive Drugs

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study involves patients with systemic sclerosis, a disease where the skin and internal organs become thick and hard due to an overproduction of connective tissue. The study focuses on patients whose disease remains active or is getting worse despite treatment with drugs that suppress the immune system, including medications such as methotrexate, azathioprine, mycophenolate mofetil, rituximab, or tocilizumab. The treatment being tested is called MB-CART19.1, which consists of the patient's own immune cells called T-cells that have been removed from the body, modified in a laboratory to target a specific protein called CD19 found on certain immune cells, and then given back to the patient through an intravenous infusion, which means the treatment is delivered directly into a vein.

The purpose of this study is to evaluate whether this modified cell therapy can improve skin thickening and fibrosis in patients with systemic sclerosis who have not responded adequately to standard treatments. The study will measure changes in skin thickness using a scoring system and will also look at other aspects of the disease, including lung function, heart function, and overall quality of life.

During the study, patients will first undergo a procedure called leucapheresis to collect their T-cells from the blood. These cells will then be modified in the laboratory and given back to the patient as a single treatment. After receiving the modified cells, patients will be monitored for six months to assess how well the treatment works and to check for any side effects. The study will also track the presence and activity of these modified cells in the body over time, as well as changes in different types of immune cells.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Cell collection procedure
Your own immune cells will be collected through a process called leukapheresis. This is a procedure where blood is drawn from your vein, specific white blood cells are separated and collected, and the remaining blood is returned to your body.
Before this procedure, you will need to stop taking certain medications. If you are taking standard immune-suppressing drugs (such as methotrexate, mycophenolate mofetil, or azathioprine), these must be stopped for 6 weeks prior to the cell collection.
If you are receiving biological treatments, a longer waiting period is required: 3 months after tocilizumab treatment, or 6 months after rituximab treatment.
Adequate vein access will be required for this procedure.
Step 2
Cell modification in laboratory
After collection, your cells will be sent to a specialized laboratory where they will be modified. The cells will be engineered to express a specific receptor that targets CD19, a protein found on certain immune cells.
This process involves using a viral vector to introduce new genetic material into your cells, creating what are called CAR T-cells (chimeric antigen receptor T-cells).
This step takes place outside your body and does not require your presence.
Step 3
Infusion of modified cells
Once the modified cells are ready, they will be given back to you through an intravenous infusion (through a vein).
The product is called MB-CART19.1 and consists of your own modified T-cells.
The infusion will be administered as a single treatment session.
Step 4
Follow-up assessments
After receiving the modified cells, you will undergo regular monitoring and assessments.
The main evaluation will occur at month 6 after the infusion, where your skin thickness will be measured using the modified Rodnan Skin Score (a standardized method of assessing skin tightness in different body areas).
Additional assessments will measure disease activity using the EUSTAR activity index, which evaluates various aspects of systemic sclerosis.
Your lung function will be tested through measurements of forced vital capacity (the amount of air you can forcefully exhale) and DLCO (a measure of how well oxygen passes from your lungs into your blood).
Imaging scans of your lungs will be performed to assess any changes in tissue scarring.
Your heart function will be evaluated through measurements of how well your heart pumps blood.
You will complete questionnaires about your ability to perform daily activities and your overall health status, including the Scleroderma Health Assessment Questionnaire.
Throughout the study, any side effects or adverse reactions will be carefully monitored and recorded.
Blood samples will be taken at various time points to measure the levels of modified cells in your body and to assess changes in your immune cell populations.

Who can join the trial?

7 criteria

You must have a diagnosis of systemic sclerosis, which is a disease where the skin and internal organs become thick and hard. This diagnosis must be confirmed according to specific medical classification systems called ACR/EULAR.
Your disease must be refractory, which means it has not responded well to previous treatments. This includes having active disease or worsening disease even after trying at least 2 different types of medicines for 6 months. These medicines include drugs called DMARDs (such as methotrexate, azathioprine, or mycophenolate mofetil) and biological DMARDs (such as rituximab or tocilizumab).
You must be between 18 and 65 years old.
Your organs must be working well enough to participate. This will be checked through tests done within 4 weeks before joining the study.
You must have suitable veins that allow doctors to perform a procedure called apheresis, which is a process to collect certain cells from your blood.
Before the cell collection procedure, you must stop taking certain immunosuppressive medicines (medicines that calm down your immune system) like methotrexate or mycophenolate mofetil for at least 6 weeks.
Before the cell collection procedure, you must have stopped biologic treatments like tocilizumab for at least 3 months, or rituximab for at least 6 months.

Who cannot join the trial?

3 criteria

The source data does not provide specific exclusion criteria (reasons why patients cannot participate) for this clinical trial
Without detailed exclusion criteria information, it is not possible to list the conditions or situations that would prevent participation in this study
The trial involves CAR T cell therapy, which is a treatment that uses modified immune cells to target specific cells in the body, but the specific reasons for excluding patients are not documented in the provided information

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Assistance Publique Hopitaux De Paris
Verified
Paris, France
Centre Hospitalier Universitaire De Lille
Verified
Lille, France
Centre Hospitalier Universitaire De Montpellier
Verified
Montpellier, France

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Investigated drugs

CAR T ANTI-CD19 is a type of cell therapy that uses the patient's own immune cells, called T-cells, which are collected from the blood and modified in a laboratory. These modified T-cells are engineered to recognize and attack cells that have a protein called CD19 on their surface. CD19 is found on certain immune cells called B-cells, which are believed to play a role in systemic sclerosis. After the T-cells are modified, they are given back to the patient through an infusion to help reduce the symptoms of the disease, particularly skin thickening and fibrosis.

Autologous T-Cells Transduced With Lentiviral Vector Expressing A Chimeric Antigen Receptor Directed Against Cd19

What is already known about the treatment

CAR T ANTI-CD19 – This is an experimental cell therapy that is currently being tested in clinical trials for treating systemic sclerosis, a disease where the immune system causes the skin and internal organs to become thick and hard. The treatment involves collecting a patient's own immune cells called T-cells, modifying them in a laboratory to recognize and destroy specific immune cells marked with a protein called CD19, and then infusing these modified cells back into the patient's body. The modified T-cells work by targeting and eliminating the B-cells that carry CD19 on their surface, which are believed to play a role in causing the abnormal immune response and tissue scarring seen in systemic sclerosis. This therapy is being studied for patients whose disease has not responded to standard treatments like steroids and other immune-suppressing medications.

Investigated diseases

Systemic Sclerosis – Systemic sclerosis is a chronic autoimmune disease that affects the connective tissue throughout the body. The condition causes the skin to become thick and hard due to excessive collagen production, a process called fibrosis. This fibrosis can also affect internal organs, particularly the lungs, heart, digestive system, and kidneys. The disease progresses differently in each patient, with some experiencing rapid worsening while others have a more stable course. Patients may develop breathing problems as lung tissue becomes scarred and stiff, reducing lung capacity. The condition can also cause the skin to tighten, limiting movement and affecting quality of life.