Scleroderma, SystemicPhase 2SCLEROCARNCT07493395
Phase IIa Trial of Anti-CD19 CAR T-Cells in Systemic Sclerosis Resistant to Immunosuppressive Therapy
A Phase 2 study testing CD19 CAR T Arm in people with Scleroderma, Systemic, run by University Hospital, Montpellier at 4 sites in France.
Trial summary
The goal of this clinical trial is to evaluate whether anti-CD19 CAR T-cell therapy can improve disease activity in adults with severe, treatment-resistant systemic sclerosis (SSc). The study will also assess the safety of this therapy and how CAR T-cells behave in the body. Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Scleroderma, Systemic
- What is being tested?
- CD19 CAR T Arm
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 4 sitesFrance
- Study dates
- 11 Jun 2026to 11 Jun 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Adequate organ functions assessed:
- serum Creatinine clearance > 40ml/mi
- adequate bone marrow function (Hemoglobin ≥9g/dL ; PMN ≥ 1 G/L ; Platelets ≥ 100 G/L)
- Alanine aminotransferase (ALT) ≤ 3 x ULN and total bilirubin < 2.0 mg/dL (34 μmol/L) (or < 3.0 mg/dL [51 μmol/L] for subjects with Gilbert's syndrome)
- Adequate respiratory function: no dyspnea or grade I dyspnea (Common Terminology Criteria for Adverse Events (NCI CTCAE v 5.0) and oxygen saturation >/= 92% on room air
- Adequate venous access for apheresis
- Leucapheresis : a wash-out period of 6 weeks for conventional immunosuppressants (i.e. methotrexate, mycophenolate mofetil)
- Leucapheresis : at least 12 weeks after biotherapy (i.e. tocilizumab, 6 months for rituximab),
Who cannot join
- Craniocerebral trauma, conscious disturbance, epilepsy, cerebrovascular ischemia or cerebrovascular hemorrhagic diseases
- ECG showing prolonged QT interval or history of severe heart diseases or FEVG < 40%
- Lung and / or heart severe dysfunction defined by CVF<50% and/or DLCO <40%
- Pulmonary arterial hypertension defined by catheterism (mean AP > 25mmHg at rest or > 30mmHg after exercise, PAOP < 15mmHG)
- Clinically significant active, opportunistic, chronic or recurrent infection (including but not limited to: hepatitis B or C virus or HIV) or covid-19 < 1 months including active or latent tuberculosis (TB) infection
- Contra indication for autologous hematopoietic stem cell transplantation (AHSCT ) or relapsing at least one year after AHSCT
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- Active hematological or solid neoplasm
- Concurrent therapy with systemic steroids (>10 mg/d prednisone equivalent) within 2 weeks prior to inclusion, except inhaled steroids
- Methylprednisolone or prednisone (maximum dose 20 mg) instead of immunosuppressive agents
- T cell targeting drugs (e.g. mycophenolate mofetil, azathioprine, calcineurin inhibitors) within 6 weeks prior to leukapheresis
- Previous adoptive T cell therapy or any gene therapy including CAR T cell therapy
- Live vaccines within 6 weeks prior to leukapheresis
- Hypersensitivity against any drug or its ingredients/impurities that is scheduled or likely to be given during trial participation, e.g. as part of the mandatory preparative chemotherapy or rescue medication/salvage therapies for treatment related toxicities
- patients without social security coverage;
- patients under guardianship;
- Male or female patients seeking to conceive a child
- Women of childbearing potential unless they are using a highly effective method of contraception starting from the time of enrolment and for at least 12 months following LD chemotherapy and until clearance of CAR-T cells, and sexually active male participants unwilling to use a condom. Female partners of sexually active male participants must be on a highly effective form of birth control from the time of enrolment and for at least 12 months following LD chemotherapy and until clearance of CAR-T cells.
- pregnant or breastfeeding women;
- patients with advanced cognitive disorders or any other cause preventing their informed consent;
- active, clinically significant CNS pathology : If signs or symptoms exist which present diagnostic uncertainty, neurologist consultation will be obtained to confirm the diagnosis of any neurological condition
- any comorbidity, whatever it may be, which may, in the opinion of the investigator, place the patient at additional risk or interfere with the monitoring of the study.
- Concurrent participation in any other interventional trial and Contraindication to the lymphodepleting chemotherapy
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
France4 sites, 4 recruiting
- Not yet recruiting
CHRU Lille
Lille
David LAUNAY, Pr, david.launay@chu-lille.fr
Ibrahim YAKOUB-AGHA, Pr
- Recruiting
Montpellier University Hospital
Montpellier
Charlotte KAAN
Christian JORGENSEN, Pr, 04 67 33 48 53 ext. 33, christian.jorgensen@inserm.fr
- Not yet recruiting
APHP Necker
Paris
Yannick ALLANORE, Pr, yannick.allanore@me.com
Olivier HERMINE, Pr
- Not yet recruiting
Chu Rouen
Rouen
Olivier BOYER, PUPH, olivier.boyer@chu-rouen.fr
About this study
Official title: SCLEROCAR: A Phase IIa Trial Evaluating the Efficacy of Anti-CD19 Chimeric Antigen Receptor Engineered T-Cells in Patients With Systemic Sclerosis (SSc) Resistant to Immunosuppressive Drugs
The goal of this clinical trial is to evaluate whether anti-CD19 CAR T-cell therapy can improve disease activity in adults with severe, treatment-resistant systemic sclerosis (SSc). The study will also assess the safety of this therapy and how CAR T-cells behave in the body.
The main questions are:
Read more
Does CAR T-cell therapy reduce skin thickening and other signs of SSc? What side effects occur after receiving CAR T-cells? How do CAR T-cells expand, persist, and affect B-cells and autoantibodies?
Participants will:
Undergo leukapheresis Receive short lymphodepleting chemotherapy Receive one infusion of anti-CD19 CAR T-cells Stay in the hospital for about 10 days Attend follow-up visits for 24 months with clinical exams, blood tests, and organ-function assessments
Optional skin or lymph-node biopsies may be performed in participants who consent to these procedures.
This study aims to provide early evidence on whether CAR T-cell therapy could become a promising treatment option for systemic sclerosis.
Read the detailed description
Systemic sclerosis (SSc) is a rare and severe autoimmune disease characterized by fibrosis of the skin and multiple organs, vasculopathy, and immune dysregulation. Many patients continue to experience active and progressive disease despite conventional immunosuppressive treatments, including disease modifying antirheumatic drugs (DMARDs) and biologics. Therapeutic options remain limited, and there is a significant unmet medical need for innovative approaches targeting the underlying mechanisms of the disease.
Recent preliminary experience from studies conducted in autoimmune diseases-such as lupus, myositis, and systemic sclerosis-suggests that autologous anti CD19 CAR T cell therapy may help reduce disease activity. Reported observations include transient B cell depletion, decreases in autoantibody levels, and improvements in joint, pulmonary, and cardiac manifestations. These early results support further evaluation of CAR T cell therapy in systemic sclerosis in a structured clinical trial setting.
This Phase IIa, multicenter, single-arm study is designed to evaluate the feasibility and safety of autologous anti-CD19 CAR-T cell therapy in adults with active systemic sclerosis who are resistant to immunosuppressive treatments. The study also aims to generate early clinical and biological data to assess the potential value of this therapeutic strategy in this population.
Eligible participants will undergo leukapheresis for T-cell collection. Following standard manufacturing of autologous anti-CD19 CAR-T cells in a GMP (Good Manufacturing Practice)-certified facility, participants will receive a short course of lymphodepleting chemotherapy, followed by a single intravenous infusion of CAR-T cells. They will remain hospitalized for intensive monitoring during the early post-infusion period, in accordance with established procedures for CAR-T cell therapies. Participants with known hypersensitivity to drugs required for treatment-related toxicity are excluded, as specified in the protocol.
After discharge from the hospital, participants will have follow-up visits for up to 24 months. These visits will include clinical and biological assessments as well as appropriate imaging tests to monitor disease activity, treatment safety, and overall health status. Biological samples will also be collected at specific times for centralized analysis.
The study incorporates exploratory immunological and translational research components. These may include monitoring circulating CAR T cells, characterizing immune cell subsets, and evaluating selected biomarkers. Optional skin and lymph node biopsies may be performed in consenting participants to analyze immune cell phenotypes and tissue level changes associated with treatment. Additional exploratory analyses may assess the spatial organization and distribution of immune cells within tissue samples, as described in the study protocol.
Overall, the study aims to provide early data on the use of anti CD19 CAR T cell therapy in systemic sclerosis, including feasibility, safety, and biological signals of activity. The results are expected to contribute to the development of innovative cell based therapeutic strategies for this severe autoimmune disease.
- Conditions
- Scleroderma, Systemic
- Being tested
- CD19 CAR T Arm
- Keywords
- Systemic Sclerosis, Anti-CD19 CAR T-cells, Autologous CAR T-cell therapy
- Medical subject headings
- Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD19 CAR T ArmBiological
Autologous anti-CD19 CAR-T cells are generated from the participant's leukapheresis product in a Good Manufacturing Practice (GMP)-certified facility using a lentiviral vector.
Prior to infusion, participants will receive a short course of lymphodepleting chemotherapy.
A single intravenous infusion of autologous anti-CD19 CAR-T cells will be administered on Day 0 at a target dose of 1 × 10⁶ CAR-T cells/kg. Participants will then be monitored in the hospital in accordance with standard post-CAR-T cell infusion procedures.
| Study group | Role | What they receive |
|---|---|---|
| CD19 CAR T Arm | Experimental | Participants assigned to this arm will undergo leukapheresis and receive a lymphodepleting chemotherapy regimen, followed by a single intravenous infusion of autologous anti-CD19 CAR T-cells on Day 0. All participants enrolled in the study are included in this single experimental arm Receives: Biological: CD19 CAR T Arm |
What is being measured
The primary outcome is the main question the study is designed to answer.
modified Rodnan skin score (mRSS)
Clinical response will be assessed through the modified Rodnan skin score (mRSS) which measures skin thickness on a scale of 0 to 3 at 17 anatomical sites (score range 0-51, higher scores indicating worse skin fibrosis)
Measured: 6 Months
Secondary outcomes (122)
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Clinical response will also be assessed through the European Scleroderma Trial And Research (EUSTAR) disease activity (range 0-10, higher scores indicating greater disease activity) based on a weighted 10-point activity index : Δ-skin=1.5 (Δ=patient assessed worsening during the previous month), modified Rodnan skin score (mRss) >18=1.5, digital ulcers=1.5, tendon friction rubs=2.25, C-reactive protein >1 mg/dL=2.25 and diffusing capacity of the lung for CO (DLCO) % predicted <70%=1.0. A cut-off ≥2.5 was found to identify patients with active disease
Measured: From 3 months to 1 month before CAR-T cell infusion
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Clinical response will also be assessed through the European Scleroderma Trial And Research (EUSTAR) disease activity (range 0-10, higher scores indicating greater disease activity) based on a weighted 10-point activity index : Δ-skin=1.5 (Δ=patient assessed worsening during the previous month), modified Rodnan skin score (mRss) >18=1.5, digital ulcers=1.5, tendon friction rubs=2.25, C-reactive protein >1 mg/dL=2.25 and diffusing capacity of the lung for CO (DLCO) % predicted <70%=1.0. A cut-off ≥2.5 was found to identify patients with active disease
Measured: 1 month before CAR-T cell infusion
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Clinical response will also be assessed through the European Scleroderma Trial And Research (EUSTAR) disease activity (range 0-10, higher scores indicating greater disease activity) based on a weighted 10-point activity index : Δ-skin=1.5 (Δ=patient assessed worsening during the previous month), modified Rodnan skin score (mRss) >18=1.5, digital ulcers=1.5, tendon friction rubs=2.25, C-reactive protein >1 mg/dL=2.25 and diffusing capacity of the lung for CO (DLCO) % predicted <70%=1.0. A cut-off ≥2.5 was found to identify patients with active disease
Measured: Day 0 (CAR-T cell infusion)
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Clinical response will also be assessed through the European Scleroderma Trial And Research (EUSTAR) disease activity (range 0-10, higher scores indicating greater disease activity) based on a weighted 10-point activity index : Δ-skin=1.5 (Δ=patient assessed worsening during the previous month), modified Rodnan skin score (mRss) >18=1.5, digital ulcers=1.5, tendon friction rubs=2.25, C-reactive protein >1 mg/dL=2.25 and diffusing capacity of the lung for CO (DLCO) % predicted <70%=1.0. A cut-off ≥2.5 was found to identify patients with active disease
Measured: Day 28 after CAR-T cell infusion
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Clinical response will also be assessed through the European Scleroderma Trial And Research (EUSTAR) disease activity (range 0-10, higher scores indicating greater disease activity) based on a weighted 10-point activity index : Δ-skin=1.5 (Δ=patient assessed worsening during the previous month), modified Rodnan skin score (mRss) >18=1.5, digital ulcers=1.5, tendon friction rubs=2.25, C-reactive protein >1 mg/dL=2.25 and diffusing capacity of the lung for CO (DLCO) % predicted <70%=1.0. A cut-off ≥2.5 was found to identify patients with active disease
Measured: 3 Months
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Clinical response will also be assessed through the European Scleroderma Trial And Research (EUSTAR) disease activity (range 0-10, higher scores indicating greater disease activity) based on a weighted 10-point activity index : Δ-skin=1.5 (Δ=patient assessed worsening during the previous month), modified Rodnan skin score (mRss) >18=1.5, digital ulcers=1.5, tendon friction rubs=2.25, C-reactive protein >1 mg/dL=2.25 and diffusing capacity of the lung for CO (DLCO) % predicted <70%=1.0. A cut-off ≥2.5 was found to identify patients with active disease
Measured: 6 Months
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Clinical response will also be assessed through the European Scleroderma Trial And Research (EUSTAR) disease activity (range 0-10, higher scores indicating greater disease activity) based on a weighted 10-point activity index : Δ-skin=1.5 (Δ=patient assessed worsening during the previous month), modified Rodnan skin score (mRss) >18=1.5, digital ulcers=1.5, tendon friction rubs=2.25, C-reactive protein >1 mg/dL=2.25 and diffusing capacity of the lung for CO (DLCO) % predicted <70%=1.0. A cut-off ≥2.5 was found to identify patients with active disease
Measured: 12 Months
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Clinical response will also be assessed through the European Scleroderma Trial And Research (EUSTAR) disease activity (range 0-10, higher scores indicating greater disease activity) based on a weighted 10-point activity index : Δ-skin=1.5 (Δ=patient assessed worsening during the previous month), modified Rodnan skin score (mRss) >18=1.5, digital ulcers=1.5, tendon friction rubs=2.25, C-reactive protein >1 mg/dL=2.25 and diffusing capacity of the lung for CO (DLCO) % predicted <70%=1.0. A cut-off ≥2.5 was found to identify patients with active disease
Measured: 24 Months
modified Rodnan skin score (mRSS)
Clinical response will be assessed through the modified Rodnan skin score (mRSS) which measures skin thickness on a scale of 0 to 3 at 17 anatomical sites (score range 0-51, higher scores indicating worse skin fibrosis)
Measured: From 3 months to 1 month before CAR-T infusion
modified Rodnan skin score (mRSS)
Clinical response will be assessed through the modified Rodnan skin score (mRSS) which measures skin thickness on a scale of 0 to 3 at 17 anatomical sites (score range 0-51, higher scores indicating worse skin fibrosis)
Measured: 1 month before CAR-T infusion
modified Rodnan skin score (mRSS)
Clinical response will be assessed through the modified Rodnan skin score (mRSS) which measures skin thickness on a scale of 0 to 3 at 17 anatomical sites (score range 0-51, higher scores indicating worse skin fibrosis)
Measured: Day 0 (CAR-T infusion)
modified Rodnan skin score (mRSS)
Clinical response will be assessed through the modified Rodnan skin score (mRSS) which measures skin thickness on a scale of 0 to 3 at 17 anatomical sites (score range 0-51, higher scores indicating worse skin fibrosis)
Measured: Day 28 after infusion
modified Rodnan skin score (mRSS)
Clinical response will be assessed through the modified Rodnan skin score (mRSS) which measures skin thickness on a scale of 0 to 3 at 17 anatomical sites (score range 0-51, higher scores indicating worse skin fibrosis)
Measured: 2 Months
modified Rodnan skin score (mRSS)
Clinical response will be assessed through the modified Rodnan skin score (mRSS) which measures skin thickness on a scale of 0 to 3 at 17 anatomical sites (score range 0-51, higher scores indicating worse skin fibrosis)
Measured: 3 Months
modified Rodnan skin score (mRSS)
Clinical response will be assessed through the modified Rodnan skin score (mRSS) which measures skin thickness on a scale of 0 to 3 at 17 anatomical sites (score range 0-51, higher scores indicating worse skin fibrosis)
Measured: 6 Months
modified Rodnan skin score (mRSS)
Clinical response will be assessed through the modified Rodnan skin score (mRSS) which measures skin thickness on a scale of 0 to 3 at 17 anatomical sites (score range 0-51, higher scores indicating worse skin fibrosis)
Measured: 12 months
modified Rodnan skin score (mRSS)
Clinical response will be assessed through the modified Rodnan skin score (mRSS) which measures skin thickness on a scale of 0 to 3 at 17 anatomical sites (score range 0-51, higher scores indicating worse skin fibrosis)
Measured: 24 months
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Clinical response will be assessed using the revised Combined Response Index in diffuse cutaneous Systemic Sclerosis (rCRISS).
The rCRISS is a 2-step composite score:
Step 1: Patients are considered not improved if any of the following occur: scleroderma renal crisis, significant FVC decline (≥15% or FVC% <80%), new left ventricular failure (LVEF ≤45%), new pulmonary arterial hypertension requiring treatment, gastrointestinal dysmotility requiring nutritional support, or digital ischemia requiring treatment or hospitalization.
Step 2: For other patients, improvement is defined as ≥40% improvement in at least 3 of 5 measures: mRSS, HAQ-DI, patient global assessment, clinician global assessment, and percent predicted FVC.
Additional assessments include spirometry (FVC, DLCO), thoracic CT fibrosis, echocardiography (LVEF, global longitudinal strain), cardiac MRI, and SHAQ score
Measured: From 3 months to 1 month before CAR-T infusion
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Clinical response will be assessed using the revised Combined Response Index in diffuse cutaneous Systemic Sclerosis (rCRISS).
The rCRISS is a 2-step composite score:
Step 1: Patients are considered not improved if any of the following occur: scleroderma renal crisis, significant FVC decline (≥15% or FVC% <80%), new left ventricular failure (LVEF ≤45%), new pulmonary arterial hypertension requiring treatment, gastrointestinal dysmotility requiring nutritional support, or digital ischemia requiring treatment or hospitalization.
Step 2: For other patients, improvement is defined as ≥40% improvement in at least 3 of 5 measures: mRSS, HAQ-DI, patient global assessment, clinician global assessment, and percent predicted FVC.
Additional assessments include spirometry (FVC, DLCO), thoracic CT fibrosis, echocardiography (LVEF, global longitudinal strain), cardiac MRI, and SHAQ score
Measured: 1 month before CAR-T infusion
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Clinical response will be assessed using the revised Combined Response Index in diffuse cutaneous Systemic Sclerosis (rCRISS).
The rCRISS is a 2-step composite score:
Step 1: Patients are considered not improved if any of the following occur: scleroderma renal crisis, significant FVC decline (≥15% or FVC% <80%), new left ventricular failure (LVEF ≤45%), new pulmonary arterial hypertension requiring treatment, gastrointestinal dysmotility requiring nutritional support, or digital ischemia requiring treatment or hospitalization.
Step 2: For other patients, improvement is defined as ≥40% improvement in at least 3 of 5 measures: mRSS, HAQ-DI, patient global assessment, clinician global assessment, and percent predicted FVC.
Additional assessments include spirometry (FVC, DLCO), thoracic CT fibrosis, echocardiography (LVEF, global longitudinal strain), cardiac MRI, and SHAQ score
Measured: Day 0 (CAR-T infusion)
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Clinical response will be assessed using the revised Combined Response Index in diffuse cutaneous Systemic Sclerosis (rCRISS).
The rCRISS is a 2-step composite score:
Step 1: Patients are considered not improved if any of the following occur: scleroderma renal crisis, significant FVC decline (≥15% or FVC% <80%), new left ventricular failure (LVEF ≤45%), new pulmonary arterial hypertension requiring treatment, gastrointestinal dysmotility requiring nutritional support, or digital ischemia requiring treatment or hospitalization.
Step 2: For other patients, improvement is defined as ≥40% improvement in at least 3 of 5 measures: mRSS, HAQ-DI, patient global assessment, clinician global assessment, and percent predicted FVC.
Additional assessments include spirometry (FVC, DLCO), thoracic CT fibrosis, echocardiography (LVEF, global longitudinal strain), cardiac MRI, and SHAQ score
Measured: Day 28 after infusion
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Clinical response will be assessed using the revised Combined Response Index in diffuse cutaneous Systemic Sclerosis (rCRISS).
The rCRISS is a 2-step composite score:
Step 1: Patients are considered not improved if any of the following occur: scleroderma renal crisis, significant FVC decline (≥15% or FVC% <80%), new left ventricular failure (LVEF ≤45%), new pulmonary arterial hypertension requiring treatment, gastrointestinal dysmotility requiring nutritional support, or digital ischemia requiring treatment or hospitalization.
Step 2: For other patients, improvement is defined as ≥40% improvement in at least 3 of 5 measures: mRSS, HAQ-DI, patient global assessment, clinician global assessment, and percent predicted FVC.
Additional assessments include spirometry (FVC, DLCO), thoracic CT fibrosis, echocardiography (LVEF, global longitudinal strain), cardiac MRI, and SHAQ score
Measured: 2 Months
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Clinical response will be assessed using the revised Combined Response Index in diffuse cutaneous Systemic Sclerosis (rCRISS).
The rCRISS is a 2-step composite score:
Step 1: Patients are considered not improved if any of the following occur: scleroderma renal crisis, significant FVC decline (≥15% or FVC% <80%), new left ventricular failure (LVEF ≤45%), new pulmonary arterial hypertension requiring treatment, gastrointestinal dysmotility requiring nutritional support, or digital ischemia requiring treatment or hospitalization.
Step 2: For other patients, improvement is defined as ≥40% improvement in at least 3 of 5 measures: mRSS, HAQ-DI, patient global assessment, clinician global assessment, and percent predicted FVC.
Additional assessments include spirometry (FVC, DLCO), thoracic CT fibrosis, echocardiography (LVEF, global longitudinal strain), cardiac MRI, and SHAQ score
Measured: 3 Months
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Clinical response will be assessed using the revised Combined Response Index in diffuse cutaneous Systemic Sclerosis (rCRISS).
The rCRISS is a 2-step composite score:
Step 1: Patients are considered not improved if any of the following occur: scleroderma renal crisis, significant FVC decline (≥15% or FVC% <80%), new left ventricular failure (LVEF ≤45%), new pulmonary arterial hypertension requiring treatment, gastrointestinal dysmotility requiring nutritional support, or digital ischemia requiring treatment or hospitalization.
Step 2: For other patients, improvement is defined as ≥40% improvement in at least 3 of 5 measures: mRSS, HAQ-DI, patient global assessment, clinician global assessment, and percent predicted FVC.
Additional assessments include spirometry (FVC, DLCO), thoracic CT fibrosis, echocardiography (LVEF, global longitudinal strain), cardiac MRI, and SHAQ score
Measured: 6 Months
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Clinical response will be assessed using the revised Combined Response Index in diffuse cutaneous Systemic Sclerosis (rCRISS).
The rCRISS is a 2-step composite score:
Step 1: Patients are considered not improved if any of the following occur: scleroderma renal crisis, significant FVC decline (≥15% or FVC% <80%), new left ventricular failure (LVEF ≤45%), new pulmonary arterial hypertension requiring treatment, gastrointestinal dysmotility requiring nutritional support, or digital ischemia requiring treatment or hospitalization.
Step 2: For other patients, improvement is defined as ≥40% improvement in at least 3 of 5 measures: mRSS, HAQ-DI, patient global assessment, clinician global assessment, and percent predicted FVC.
Additional assessments include spirometry (FVC, DLCO), thoracic CT fibrosis, echocardiography (LVEF, global longitudinal strain), cardiac MRI, and SHAQ score
Measured: 12 Months
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Clinical response will be assessed using the revised Combined Response Index in diffuse cutaneous Systemic Sclerosis (rCRISS).
The rCRISS is a 2-step composite score:
Step 1: Patients are considered not improved if any of the following occur: scleroderma renal crisis, significant FVC decline (≥15% or FVC% <80%), new left ventricular failure (LVEF ≤45%), new pulmonary arterial hypertension requiring treatment, gastrointestinal dysmotility requiring nutritional support, or digital ischemia requiring treatment or hospitalization.
Step 2: For other patients, improvement is defined as ≥40% improvement in at least 3 of 5 measures: mRSS, HAQ-DI, patient global assessment, clinician global assessment, and percent predicted FVC.
Additional assessments include spirometry (FVC, DLCO), thoracic CT fibrosis, echocardiography (LVEF, global longitudinal strain), cardiac MRI, and SHAQ score
Measured: 24 Months
Change in the lung capacity FVC (forced vital capacity)
To assess the impact of the treatment on pulmonary function, FVC will be assessed during functionnal exploration test lab and we will evaluate the improvement in FVC over time
Measured: From 3 months to 1 month before CAR-T infusion
Change in the lung capacity FVC (forced vital capacity)
To assess the impact of the treatment on pulmonary function, FVC will be assessed during functionnal exploration test lab and we will evaluate the improvement in FVC over time
Measured: 1 month before CAR-T infusion
Change in the lung capacity FVC (forced vital capacity)
To assess the impact of the treatment on pulmonary function, FVC will be assessed during functionnal exploration test lab and we will evaluate the improvement in FVC over time
Measured: 3 Months
Change in the lung capacity FVC (forced vital capacity)
To assess the impact of the treatment on pulmonary function, FVC will be assessed during functionnal exploration test lab and we will evaluate the improvement in FVC over time
Measured: 6 Months
Change in the lung capacity FVC (forced vital capacity)
To assess the impact of the treatment on pulmonary function, FVC will be assessed during functionnal exploration test lab and we will evaluate the improvement in FVC over time
Measured: 12 Months
Change in the lung capacity FVC (forced vital capacity)
To assess the impact of the treatment on pulmonary function, FVC will be assessed during functionnal exploration test lab and we will evaluate the improvement in FVC over time
Measured: 24 Months
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
The efficacy of the treatment on pulmonary function will also be analyzed by monitoring changes in DLCO (diffusing capacity of the lung for carbon monoxide), quantifiy during functionnal exploration test lab
Measured: From 3 months to 1 month before CAR-T infusion
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
The efficacy of the treatment on pulmonary function will also be analyzed by monitoring changes in DLCO (diffusing capacity of the lung for carbon monoxide), quantifiy during functionnal exploration test lab
Measured: 1 month before CAR-T infusion
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
The efficacy of the treatment on pulmonary function will also be analyzed by monitoring changes in DLCO (diffusing capacity of the lung for carbon monoxide), quantifiy during functionnal exploration test lab
Measured: 3 Months
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
The efficacy of the treatment on pulmonary function will also be analyzed by monitoring changes in DLCO (diffusing capacity of the lung for carbon monoxide), quantifiy during functionnal exploration test lab
Measured: 6 Months
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
The efficacy of the treatment on pulmonary function will also be analyzed by monitoring changes in DLCO (diffusing capacity of the lung for carbon monoxide), quantifiy during functionnal exploration test lab
Measured: 12 Months
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
The efficacy of the treatment on pulmonary function will also be analyzed by monitoring changes in DLCO (diffusing capacity of the lung for carbon monoxide), quantifiy during functionnal exploration test lab
Measured: 24 Months
Extent of fibrosis on pulmonary CT (Computed Tomography)
Pulmonary response will be also assessment by CT scan and expressed as percentage of parenchyma affected. The type of parenchymal involvement willbe specified (ground glass, honeycomb)
Measured: From 3 months to 1 month before CAR-T infusion
Extent of fibrosis on pulmonary CT(Computed Tomography)
Pulmonary response will be also assessment by CT scan and expressed as percentage of parenchyma affected. The type of parenchymal involvement willbe specified (ground glass, honeycomb)
Measured: 3 Months
Extent of fibrosis on pulmonary CT(Computed Tomography)
Pulmonary response will be also assessment by CT scan and expressed as percentage of parenchyma affected. The type of parenchymal involvement willbe specified (ground glass, honeycomb)
Measured: 12 Months
Extent of fibrosis on pulmonary CT(Computed Tomography)
Pulmonary response will be also assessment by CT scan and expressed as percentage of parenchyma affected. The type of parenchymal involvement willbe specified (ground glass, honeycomb)
Measured: 24 Months
Change in cardiac ejection fraction and global longitudinal strain
A transthoracic cardiac ultrasound will be performed regularly to assess the effect of treatment on the left ventricular ejection fraction, as well as the global longitudinal strain (GLS).
Measured: From 3 months to 1 month before CAR-T infusion
Change in cardiac ejection fraction and global longitudinal strain
A transthoracic cardiac ultrasound will be performed regularly to assess the effect of treatment on the left ventricular ejection fraction, as well as the global longitudinal strain (GLS).
Measured: 3 Months
Change in cardiac ejection fraction and global longitudinal strain
A transthoracic cardiac ultrasound will be performed regularly to assess the effect of treatment on the left ventricular ejection fraction, as well as the global longitudinal strain (GLS).
Measured: 12 Months
Change in cardiac ejection fraction and global longitudinal strain
A transthoracic cardiac ultrasound will be performed regularly to assess the effect of treatment on the left ventricular ejection fraction, as well as the global longitudinal strain (GLS).
Measured: 24 Months
Cardiomyopathy, change in cardiac MRI (Magnetic Resonance Imaging) signal
A cardiac MRI (Magnetic Resonance Imaging) will be performed at baseline and at follow-up visit to evaluate the effect of treatment on T1 and T2 mapping, late gadolinium enhancement (LGE), extracellular volume ( ECV) mapping and indirect indicators of pulmonary hypertension
Measured: From 3 months to 1 month before CAR-T infusion
Cardiomyopathy, change in cardiac MRI (Magnetic Resonance Imaging) signal
A cardiac MRI (Magnetic Resonance Imaging) will be performed at baseline and at follow-up visit to evaluate the effect of treatment on T1 and T2 mapping, late gadolinium enhancement (LGE), extracellular volume ( ECV) mapping and indirect indicators of pulmonary hypertension
Measured: 12 Months
Cardiomyopathy, change in cardiac MRI (Magnetic Resonance Imaging) signal
A cardiac MRI (Magnetic Resonance Imaging) will be performed at baseline and at follow-up visit to evaluate the effect of treatment on T1 and T2 mapping, late gadolinium enhancement (LGE), extracellular volume ( ECV) mapping and indirect indicators of pulmonary hypertension
Measured: 24 Months
Change in scleroderma-adapted Scleroderma Health Assessment Questionnaire (SHAQ) score
Clinical response will be assessed using SHAQ : the patient will complete a self-assessment questionnaire on their health status during systemic sclerosis (SSc) and we will compare the scores at each stage
Measured: Day 0 (CAR-T infusion)
Change in scleroderma-adapted Scleroderma Health Assessment Questionnaire (SHAQ) score
Clinical response will be assessed using SHAQ : the patient will complete a self-assessment questionnaire on their health status during systemic sclerosis (SSc) and we will compare the scores at each stage
Measured: Day 28 after CAR-T cell infusion
Change in scleroderma-adapted Scleroderma Health Assessment Questionnaire (SHAQ) score
Clinical response will be assessed using SHAQ : the patient will complete a self-assessment questionnaire on their health status during systemic sclerosis (SSc) and we will compare the scores at each stage
Measured: 3 Months
Change in scleroderma-adapted Scleroderma Health Assessment Questionnaire (SHAQ) score
Clinical response will be assessed using SHAQ : the patient will complete a self-assessment questionnaire on their health status during systemic sclerosis (SSc) and we will compare the scores at each stage
Measured: 6 Months
Change in scleroderma-adapted Scleroderma Health Assessment Questionnaire (SHAQ) score
Clinical response will be assessed using SHAQ : the patient will complete a self-assessment questionnaire on their health status during systemic sclerosis (SSc) and we will compare the scores at each stage
Measured: 12 Months
Change in scleroderma-adapted Scleroderma Health Assessment Questionnaire (SHAQ) score
Clinical response will be assessed using SHAQ : the patient will complete a self-assessment questionnaire on their health status during systemic sclerosis (SSc) and we will compare the scores at each stage
Measured: 24 Months
Change in Health Assessment Questionnaire Disability Index HAQ-DI score
The Health Assessment Questionnaire - Disability Index (HAQ-DI) is a quantitative tool used to measure health related quality of life assessments related to SSc ; Clinical response will be assessed using HAQ-DI and we will compare the scores at each stage
Measured: Day 0 (CAR-T cell infusion)
Change in Health Assessment Questionnaire Disability Index HAQ-DI score
The Health Assessment Questionnaire - Disability Index (HAQ-DI) is a quantitative tool used to measure health related quality of life assessments related to SSc ; Clinical response will be assessed using HAQ-DI and we will compare the scores at each stage
Measured: Day 28 after CAR-T cell infusion
Change in Health Assessment Questionnaire Disability Index HAQ-DI score
The Health Assessment Questionnaire - Disability Index (HAQ-DI) is a quantitative tool used to measure health related quality of life assessments related to SSc ; Clinical response will be assessed using HAQ-DI and we will compare the scores at each stage
Measured: 3 Months
Change in Health Assessment Questionnaire Disability Index HAQ-DI score
The Health Assessment Questionnaire - Disability Index (HAQ-DI) is a quantitative tool used to measure health related quality of life assessments related to SSc ; Clinical response will be assessed using HAQ-DI and we will compare the scores at each stage
Measured: 6 Months
Change in Health Assessment Questionnaire Disability Index HAQ-DI score
The Health Assessment Questionnaire - Disability Index (HAQ-DI) is a quantitative tool used to measure health related quality of life assessments related to SSc ; Clinical response will be assessed using HAQ-DI and we will compare the scores at each stage
Measured: 12 Months
Change in Health Assessment Questionnaire Disability Index HAQ-DI score
The Health Assessment Questionnaire - Disability Index (HAQ-DI) is a quantitative tool used to measure health related quality of life assessments related to SSc ; Clinical response will be assessed using HAQ-DI and we will compare the scores at each stage
Measured: 24 Months
Change in Health Assessment Questionnaire Cochin Hand score
Clinical response will also be assessed using the Cochin Hand Function Scale, a validated tool that quantifies disability resulting from hand functional impairment.
Measured: Day 0 (CAR-T cell infusion)
Change in Health Assessment Questionnaire Cochin Hand score
Clinical response will also be assessed using the Cochin Hand Function Scale, a validated tool that quantifies disability resulting from hand functional impairment.
Measured: Day 28 after CAR-T cell infusion
Change in Health Assessment Questionnaire Cochin Hand score
Clinical response will also be assessed using the Cochin Hand Function Scale, a validated tool that quantifies disability resulting from hand functional impairment.
Measured: 3 Months
Change in Health Assessment Questionnaire Cochin Hand score
Clinical response will also be assessed using the Cochin Hand Function Scale, a validated tool that quantifies disability resulting from hand functional impairment.
Measured: 6 Months
Change in Health Assessment Questionnaire Cochin Hand score
Clinical response will also be assessed using the Cochin Hand Function Scale, a validated tool that quantifies disability resulting from hand functional impairment.
Measured: 12 Months
Change in Health Assessment Questionnaire Cochin Hand score
Clinical response will also be assessed using the Cochin Hand Function Scale, a validated tool that quantifies disability resulting from hand functional impairment.
Measured: 24 Months
Change in quality of life Questionnaire SF-36
Clinical response will also be assessed using the SF36 score collected at each time point through the SF36 self-assessment questionnaire, which evaluates the patient's quality of life across eight domains, including physical functioning, role limitations, bodily pain, general health, vitality, social functioning, emotional well-being, and mental health
Measured: Day 0 (CAR-T cell infusion)
Change in quality of life Questionnaire SF-36
Clinical response will also be assessed using the SF36 score collected at each time point through the 36-Item Short Form Survey (SF-36) self-assessment questionnaire, which evaluates the patient's quality of life across eight domains, including physical functioning, role limitations, bodily pain, general health, vitality, social functioning, emotional well-being, and mental health
Measured: Day 28 after CAR-T cell infusion
Change in quality of life Questionnaire SF-36
Clinical response will also be assessed using the SF36 score collected at each time point through the 36-Item Short Form Survey (SF-36) self-assessment questionnaire, which evaluates the patient's quality of life across eight domains, including physical functioning, role limitations, bodily pain, general health, vitality, social functioning, emotional well-being, and mental health
Measured: 3 Months
Change in quality of life Questionnaire SF-36
Clinical response will also be assessed using the SF36 score collected at each time point through the 36-Item Short Form Survey (SF-36) self-assessment questionnaire, which evaluates the patient's quality of life across eight domains, including physical functioning, role limitations, bodily pain, general health, vitality, social functioning, emotional well-being, and mental health
Measured: 6 Months
Change in quality of life Questionnaire SF-36
Clinical response will also be assessed using the SF36 score collected at each time point through the 36-Item Short Form Survey (SF-36) self-assessment questionnaire, which evaluates the patient's quality of life across eight domains, including physical functioning, role limitations, bodily pain, general health, vitality, social functioning, emotional well-being, and mental health
Measured: 12 Months
Change in quality of life Questionnaire SF-36
Clinical response will also be assessed using the SF36 score collected at each time point through the 36-Item Short Form Survey (SF-36) self-assessment questionnaire, which evaluates the patient's quality of life across eight domains, including physical functioning, role limitations, bodily pain, general health, vitality, social functioning, emotional well-being, and mental health
Measured: 24 Months
Change in the university of California Los Angeles scleroderma clinical trials consortium gastrointestinal tract (ULCA-SCTC GIT) score
to evaluate disease response on to systemic sclerosis associated gastrointestinal tract symptoms severity and its impact on patients' well-being, we will compare ULCA-SCTC GIT Score, which is a self-administered questionnaire completed at each time point
Measured: Day 0 (CAR-T cell infusion)
Change in the university of California Los Angeles scleroderma clinical trials consortium gastrointestinal tract (ULCA-SCTC GIT) score
to evaluate disease response on to systemic sclerosis associated gastrointestinal tract symptoms severity and its impact on patients' well-being, we will compare ULCA-SCTC GIT Score, which is a self-administered questionnaire completed at each time point
Measured: Day 28 after CAR-T cell infusion
Change in the university of California Los Angeles scleroderma clinical trials consortium gastrointestinal tract (ULCA-SCTC GIT) score
to evaluate disease response on to systemic sclerosis associated gastrointestinal tract symptoms severity and its impact on patients' well-being, we will compare ULCA-SCTC GIT Score, which is a self-administered questionnaire completed at each time point
Measured: 3 Months
Change in the university of California Los Angeles scleroderma clinical trials consortium gastrointestinal tract (ULCA-SCTC GIT) score
to evaluate disease response on to systemic sclerosis associated gastrointestinal tract symptoms severity and its impact on patients' well-being, we will compare ULCA-SCTC GIT Score, which is a self-administered questionnaire completed at each time point
Measured: 6 Months
Change in the university of California Los Angeles scleroderma clinical trials consortium gastrointestinal tract (ULCA-SCTC GIT) score
to evaluate disease response on to systemic sclerosis associated gastrointestinal tract symptoms severity and its impact on patients' well-being, we will compare ULCA-SCTC GIT Score, which is a self-administered questionnaire completed at each time point
Measured: 12 Months
Change in the university of California Los Angeles scleroderma clinical trials consortium gastrointestinal tract (ULCA-SCTC GIT) score
to evaluate disease response on to systemic sclerosis associated gastrointestinal tract symptoms severity and its impact on patients' well-being, we will compare ULCA-SCTC GIT Score, which is a self-administered questionnaire completed at each time point
Measured: 24 Months
Change in Malnutrition Universal Screening Tool (MUST) score
We will also assess the effect of treatment on digestive impairment in scleroderma patient, as well as their risk of malnutrition, using the MUST score. This evaluation includes measuring the percentage of weight loss over the previous 3 to 6 months, calculating body mass index, and determining whether the patient has been seriously ill and experienced a period of more than 5 days without nutritional intake
Measured: From 3 months to 1 month before CAR-T cell infusion
Change in Malnutrition Universal Screening Tool (MUST) score
We will also assess the effect of treatment on digestive impairment in scleroderma patient, as well as their risk of malnutrition, using the MUST score. This evaluation includes measuring the percentage of weight loss over the previous 3 to 6 months, calculating body mass index, and determining whether the patient has been seriously ill and experienced a period of more than 5 days without nutritional intake
Measured: 1 month before CAR-T cell infusion
Change in Malnutrition Universal Screening Tool (MUST) score
We will also assess the effect of treatment on digestive impairment in scleroderma patient, as well as their risk of malnutrition, using the MUST score. This evaluation includes measuring the percentage of weight loss over the previous 3 to 6 months, calculating body mass index, and determining whether the patient has been seriously ill and experienced a period of more than 5 days without nutritional intake
Measured: Day 0 (CAR-T cell infusion)
Change in Malnutrition Universal Screening Tool (MUST) score
We will also assess the effect of treatment on digestive impairment in scleroderma patient, as well as their risk of malnutrition, using the MUST score. This evaluation includes measuring the percentage of weight loss over the previous 3 to 6 months, calculating body mass index, and determining whether the patient has been seriously ill and experienced a period of more than 5 days without nutritional intake
Measured: Day 28 after CAR-T cell infusion
Change in Malnutrition Universal Screening Tool (MUST) score
We will also assess the effect of treatment on digestive impairment in scleroderma patient, as well as their risk of malnutrition, using the MUST score. This evaluation includes measuring the percentage of weight loss over the previous 3 to 6 months, calculating body mass index, and determining whether the patient has been seriously ill and experienced a period of more than 5 days without nutritional intake
Measured: 3 Months
Change in Malnutrition Universal Screening Tool (MUST) score
We will also assess the effect of treatment on digestive impairment in scleroderma patient, as well as their risk of malnutrition, using the MUST score. This evaluation includes measuring the percentage of weight loss over the previous 3 to 6 months, calculating body mass index, and determining whether the patient has been seriously ill and experienced a period of more than 5 days without nutritional intake
Measured: 6 Months
Change in Malnutrition Universal Screening Tool (MUST) score
We will also assess the effect of treatment on digestive impairment in scleroderma patient, as well as their risk of malnutrition, using the MUST score. This evaluation includes measuring the percentage of weight loss over the previous 3 to 6 months, calculating body mass index, and determining whether the patient has been seriously ill and experienced a period of more than 5 days without nutritional intake
Measured: 12 Months
Change in Malnutrition Universal Screening Tool (MUST) score
We will also assess the effect of treatment on digestive impairment in scleroderma patient, as well as their risk of malnutrition, using the MUST score. This evaluation includes measuring the percentage of weight loss over the previous 3 to 6 months, calculating body mass index, and determining whether the patient has been seriously ill and experienced a period of more than 5 days without nutritional intake
Measured: 24 Months
Anti-antibody titers
Measurement of antinuclear antibodies (ANA), anti-centromere, anti-DNA, anti-Topoisomerase, and TRAK antibody titers following CAR T anti-CD19 cell administration
Measured: From 3 months to 1 month before CAR-T cell infusion
Anti-antibody titers
Measurement of antinuclear antibodies (ANA), anti-centromere, anti-DNA, anti-Topoisomerase, and TRAK antibody titers following CAR T anti-CD19 cell administration
Measured: Day 28 after CAR-T cell infusion
Anti-antibody titers
Measurement of antinuclear antibodies (ANA), anti-centromere, anti-DNA, anti-Topoisomerase, and TRAK antibody titers following CAR T anti-CD19 cell administration
Measured: 6 Months
Anti-antibody titers
Measurement of antinuclear antibodies (ANA), anti-centromere, anti-DNA, anti-Topoisomerase, and TRAK antibody titers following CAR T anti-CD19 cell administration
Measured: 12 Months
CAR T cell counts
Quantification of CAR T cells using flow cytometry and qPCR to assess expansion and persistence after CAR T anti-CD19 infusion
Measured: Day 0 (CAR-T cell infusion)
CAR T cell counts
Quantification of CAR T cells using flow cytometry and qPCR to assess expansion and persistence after CAR T anti-CD19 infusion
Measured: Day 4 after CAR-T cell infusion
CAR T cell counts
Quantification of CAR T cells using flow cytometry and qPCR to assess expansion and persistence after CAR T anti-CD19 infusion
Measured: Day 7 after CAR-T cell infusion
CAR T cell counts
Quantification of CAR T cells using flow cytometry and qPCR to assess expansion and persistence after CAR T anti-CD19 infusion
Measured: Day 10 after CAR-T cell infusion
CAR T cell counts
Quantification of CAR T cells using flow cytometry and qPCR to assess expansion and persistence after CAR T anti-CD19 infusion
Measured: Day 14 after CAR-T cell infusion
CAR T cell counts
Quantification of CAR T cells using flow cytometry and qPCR to assess expansion and persistence after CAR T anti-CD19 infusion
Measured: Day 21 after CAR-T cell infusion
CAR T cell counts
Quantification of CAR T cells using flow cytometry and qPCR to assess expansion and persistence after CAR T anti-CD19 infusion
Measured: Day 28 after CAR-T cell infusion
CAR T cell counts
Quantification of CAR T cells using flow cytometry and qPCR to assess expansion and persistence after CAR T anti-CD19 infusion
Measured: 3 Months
CAR T cell counts
Quantification of CAR T cells using flow cytometry and qPCR to assess expansion and persistence after CAR T anti-CD19 infusion
Measured: 6 Months
CAR T cell counts
Quantification of CAR T cells using flow cytometry and qPCR to assess expansion and persistence after CAR T anti-CD19 infusion
Measured: 12 Months
CAR T cell counts
Quantification of CAR T cells using flow cytometry and qPCR to assess expansion and persistence after CAR T anti-CD19 infusion
Measured: 24 Months
Immunophenotyping of B, T, and NK cells subsets
Immunophenotyping of B cells, T cells, NK cells, and immune subpopulations to evaluate immune reconstitution following CAR T infusion
Measured: Day -14 (14 days before CAR-T infusion)
Immunophenotyping of B, T, and NK cells subsets
Immunophenotyping of B cells, T cells, NK cells, and immune subpopulations to evaluate immune reconstitution following CAR T infusion
Measured: Day 28 after CAR-T cell infusion
Immunophenotyping of B, T, and NK cells subsets
Immunophenotyping of B cells, T cells, NK cells, and immune subpopulations to evaluate immune reconstitution following CAR T infusion
Measured: 3 Months
Immunophenotyping of B, T, and NK cells subsets
Immunophenotyping of B cells, T cells, NK cells, and immune subpopulations to evaluate immune reconstitution following CAR T infusion
Measured: 6 Months
Immunophenotyping of B, T, and NK cells subsets
Immunophenotyping of B cells, T cells, NK cells, and immune subpopulations to evaluate immune reconstitution following CAR T infusion
Measured: 12 Months
B-cell receptor (BCR) repertoire analysis
BCR repertoire analysis performed on B cells from Systemic Sclerosis (SSc) patients. Assessment occurs at Month 6 if B-cell percentage is >50% of baseline; otherwise at Month 12 after CAR T anti-CD19 administration
Measured: Day -14 (14 days before CAR-T infusion)
B-cell receptor (BCR) repertoire analysis
BCR repertoire analysis performed on B cells from Systemic Sclerosis (SSc) patients. Assessment occurs at Month 6 if B-cell percentage is >50% of baseline; otherwise at Month 12 after CAR T anti-CD19 administration
Measured: 6 Months
B-cell receptor (BCR) repertoire analysis
BCR repertoire analysis performed on B cells from Systemic Sclerosis (SSc) patients. Assessment occurs at Month 6 if B-cell percentage is >50% of baseline; otherwise at Month 12 after CAR T anti-CD19 administration
Measured: 12 Months
B-cell receptor (BCR) repertoire analysis
BCR repertoire analysis performed on B cells from Systemic Sclerosis (SSc) patients. Assessment occurs at Month 6 if B-cell percentage is >50% of baseline; otherwise at Month 12 after CAR T anti-CD19 administration
Measured: 24 Months
Skin biopsies for single-cell phenotyping
Skin biopsies (for patients who have given their consent) performed at baseline for single-cell phenotyping analyses.
Measured: From 3 months to 1 month before CAR-T infusion
Skin biopsies for single-cell phenotyping
Skin biopsies (for patients who have given their consent) performed at M3 for single-cell phenotyping analyses.
Measured: 3 Months
lymph node biopsies for single-cell phenotyping
lymph node biopsies (for patients who have given their consent) performed at Month 3 for single-cell phenotyping analyses.
Measured: 3 Months
Incidence rate of adverse events
Incidence rate of adverse events graded according to the Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0 and classified following the American Society of Clinical Oncology (ASCO) guidelines
Measured: Day -14 to 24 months
Incidence of Cytokine Release Syndrome (CRS)
Incidence and severity of Cytokine Release Syndrome (CRS), graded according to the American Society for Transplantation and Cellular Therapy (ASTCT) consensus grading system.
Measured: Day -14 to 24 months
Incidence of Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS)
Incidence and severity of Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), graded according to the American Society for Transplantation and Cellular Therapy (ASTCT) consensus grading system
Measured: Day -14 to 24 months
Incidence of Cytopenias
Incidence and severity of cytopenias, graded according to the consensus grading system of the European Hematology Association (EHA) and the European Society for Blood and Marrow Transplantation (EBMT) consensus grading system
Measured: Day -14 to 24 months
Incidence of Infections
Incidence and severity of infections, graded according to Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0.
Measured: Day -14 to 24 months
Incidence of Cardiac Events
Incidence and severity of cardiac adverse events, graded according to Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0.
Measured: Day -14 to 24 months
Incidence of Acute Kidney Injury
Incidence and severity of acute kidney injury, graded according to Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0.
Measured: Day -14 to 24 months
Incidence of Scleroderma Renal Crisis
Incidence of clinically diagnosed scleroderma renal crisis
Measured: Day -14 to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2026
- Study start11 Jun 2026
- Primary completion11 Jun 2028 (estimated)
- Study completion11 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Scleroderma, SystemicStudies of the same condition, open and under way.
- More trials run by University Hospital, MontpellierThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A phase 2a, multicenter, open-label, single-arm study to investigate the efficacy of anti-CD19 CAR T-cell therapy in patients with systemic sclerosis who are resistant to immunosuppressive therapy
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Montpellier
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Christian Jorgensen, PUPH, Principal investigator, University Hospital, Montpellier
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07493395
- Sponsor's protocol ID
- RECHMPL24_0438
- Acronym
- SCLEROCAR
- Status last verified
- Oct 2026
- First submitted
- 16 Mar 2026
- Last updated
- 14 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- SCLEROCAR: A Phase IIa Trial Evaluating the Efficacy of Anti-CD19 Chimeric Antigen Receptor Engineered T-Cells in Patients With Systemic Sclerosis (SSc) Resistant to Immunosuppressive Drugs
- ClinicalTrials.gov ID
- NCT07493395
- Acronym / sponsor ID
- SCLEROCAR / RECHMPL24_0438
- Conditions
- Scleroderma, Systemic
- Keywords
- Systemic Sclerosis, Anti-CD19 CAR T-cells, Autologous CAR T-cell therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- University Hospital, MontpellierUniversity, hospital or other
- Study start
- 11 Jun 2026 (actual)
- Primary completion
- 11 Jun 2028 (estimated)
- Study completion
- 11 Jun 2028 (estimated)
- First posted
- 25 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to evaluate whether anti-CD19 CAR T-cell therapy can improve disease activity in adults with severe, treatment-resistant systemic sclerosis (SSc). The study will also assess the safety of this therapy and how CAR T-cells behave in the body.
The main questions are:
Does CAR T-cell therapy reduce skin thickening and other signs of SSc? What side effects occur after receiving CAR T-cells? How do CAR T-cells expand, persist, and affect B-cells and autoantibodies?
Participants will:
Undergo leukapheresis Receive short lymphodepleting chemotherapy Receive one infusion of anti-CD19 CAR T-cells Stay in the hospital for about 10 days Attend follow-up visits for 24 months with clinical exams, blood tests, and organ-function assessments
Optional skin or lymph-node biopsies may be performed in participants who consent to these procedures.
This study aims to provide early evidence on whether CAR T-cell therapy could become a promising treatment option for systemic sclerosis.
Detailed description
Systemic sclerosis (SSc) is a rare and severe autoimmune disease characterized by fibrosis of the skin and multiple organs, vasculopathy, and immune dysregulation. Many patients continue to experience active and progressive disease despite conventional immunosuppressive treatments, including disease modifying antirheumatic drugs (DMARDs) and biologics. Therapeutic options remain limited, and there is a significant unmet medical need for innovative approaches targeting the underlying mechanisms of the disease.
Recent preliminary experience from studies conducted in autoimmune diseases-such as lupus, myositis, and systemic sclerosis-suggests that autologous anti CD19 CAR T cell therapy may help reduce disease activity. Reported observations include transient B cell depletion, decreases in autoantibody levels, and improvements in joint, pulmonary, and cardiac manifestations. These early results support further evaluation of CAR T cell therapy in systemic sclerosis in a structured clinical trial setting.
This Phase IIa, multicenter, single-arm study is designed to evaluate the feasibility and safety of autologous anti-CD19 CAR-T cell therapy in adults with active systemic sclerosis who are resistant to immunosuppressive treatments. The study also aims to generate early clinical and biological data to assess the potential value of this therapeutic strategy in this population.
Eligible participants will undergo leukapheresis for T-cell collection. Following standard manufacturing of autologous anti-CD19 CAR-T cells in a GMP (Good Manufacturing Practice)-certified facility, participants will receive a short course of lymphodepleting chemotherapy, followed by a single intravenous infusion of CAR-T cells. They will remain hospitalized for intensive monitoring during the early post-infusion period, in accordance with established procedures for CAR-T cell therapies. Participants with known hypersensitivity to drugs required for treatment-related toxicity are excluded, as specified in the protocol.
After discharge from the hospital, participants will have follow-up visits for up to 24 months. These visits will include clinical and biological assessments as well as appropriate imaging tests to monitor disease activity, treatment safety, and overall health status. Biological samples will also be collected at specific times for centralized analysis.
The study incorporates exploratory immunological and translational research components. These may include monitoring circulating CAR T cells, characterizing immune cell subsets, and evaluating selected biomarkers. Optional skin and lymph node biopsies may be performed in consenting participants to analyze immune cell phenotypes and tissue level changes associated with treatment. Additional exploratory analyses may assess the spatial organization and distribution of immune cells within tissue samples, as described in the study protocol.
Overall, the study aims to provide early data on the use of anti CD19 CAR T cell therapy in systemic sclerosis, including feasibility, safety, and biological signals of activity. The results are expected to contribute to the development of innovative cell based therapeutic strategies for this severe autoimmune disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A phase 2a, multicenter, open-label, single-arm study to investigate the efficacy of anti-CD19 CAR T-cell therapy in patients with systemic sclerosis who are resistant to immunosuppressive therapy
| Arm | Type | Receives |
|---|---|---|
| CD19 CAR T Arm | Experimental | Biological: CD19 CAR T ArmParticipants assigned to this arm will undergo leukapheresis and receive a lymphodepleting chemotherapy regimen, followed by a single intravenous infusion of autologous anti-CD19 CAR T-cells on Day 0. All participants enrolled in the study are included in this single experimental arm |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- Adequate organ functions assessed:
- Adequate venous access for apheresis
- Leucapheresis : a wash-out period of 6 weeks for conventional immunosuppressants (i.e. methotrexate, mycophenolate mofetil)
- Leucapheresis : at least 12 weeks after biotherapy (i.e. tocilizumab, 6 months for rituximab),
Key exclusion criteria (22 listed)
- Craniocerebral trauma, conscious disturbance, epilepsy, cerebrovascular ischemia or cerebrovascular hemorrhagic diseases
- ECG showing prolonged QT interval or history of severe heart diseases or FEVG < 40%
- Lung and / or heart severe dysfunction defined by CVF<50% and/or DLCO <40%
- Pulmonary arterial hypertension defined by catheterism (mean AP > 25mmHg at rest or > 30mmHg after exercise, PAOP < 15mmHG)
- Clinically significant active, opportunistic, chronic or recurrent infection (including but not limited to: hepatitis B or C virus or HIV) or covid-19 < 1 months including active or latent tuberculosis (TB) infection
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CD19 CAR T ArmAutologous anti-CD19 CAR-T cells are generated from the participant's leukapheresis product in a Good Manufacturing Practice (GMP)-certified facility using a lentiviral vector. Prior to infusion, participants will receive a short course of lymphodepleting chemotherapy. A single intravenous infusion of autologous anti-CD19 CAR-T cells will be administered on Day 0 at a target dose of 1 × 10⁶ CAR-T cells/kg. Participants will then be monitored in the hospital in accordance with standard post-CAR-T cell infusion procedures. | Biological | CD19 CAR T Arm |
Primary outcome
modified Rodnan skin score (mRSS)
Time frame: 6 Months
Clinical response will be assessed through the modified Rodnan skin score (mRSS) which measures skin thickness on a scale of 0 to 3 at 17 anatomical sites (score range 0-51, higher scores indicating worse skin fibrosis)
122 secondary outcomes
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Time frame: From 3 months to 1 month before CAR-T cell infusion
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Time frame: 1 month before CAR-T cell infusion
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Time frame: Day 0 (CAR-T cell infusion)
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Time frame: Day 28 after CAR-T cell infusion
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Time frame: 3 Months
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Time frame: 6 Months
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Time frame: 12 Months
Change in European Scleroderma Trial And Research (EUSTAR) activity index
Time frame: 24 Months
modified Rodnan skin score (mRSS)
Time frame: From 3 months to 1 month before CAR-T infusion
modified Rodnan skin score (mRSS)
Time frame: 1 month before CAR-T infusion
modified Rodnan skin score (mRSS)
Time frame: Day 0 (CAR-T infusion)
modified Rodnan skin score (mRSS)
Time frame: Day 28 after infusion
modified Rodnan skin score (mRSS)
Time frame: 2 Months
modified Rodnan skin score (mRSS)
Time frame: 3 Months
modified Rodnan skin score (mRSS)
Time frame: 6 Months
modified Rodnan skin score (mRSS)
Time frame: 12 months
modified Rodnan skin score (mRSS)
Time frame: 24 months
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Time frame: From 3 months to 1 month before CAR-T infusion
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Time frame: 1 month before CAR-T infusion
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Time frame: Day 0 (CAR-T infusion)
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Time frame: Day 28 after infusion
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Time frame: 2 Months
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Time frame: 3 Months
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Time frame: 6 Months
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Time frame: 12 Months
Change in Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS)
Time frame: 24 Months
Change in the lung capacity FVC (forced vital capacity)
Time frame: From 3 months to 1 month before CAR-T infusion
Change in the lung capacity FVC (forced vital capacity)
Time frame: 1 month before CAR-T infusion
Change in the lung capacity FVC (forced vital capacity)
Time frame: 3 Months
Change in the lung capacity FVC (forced vital capacity)
Time frame: 6 Months
Change in the lung capacity FVC (forced vital capacity)
Time frame: 12 Months
Change in the lung capacity FVC (forced vital capacity)
Time frame: 24 Months
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
Time frame: From 3 months to 1 month before CAR-T infusion
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
Time frame: 1 month before CAR-T infusion
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
Time frame: 3 Months
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
Time frame: 6 Months
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
Time frame: 12 Months
Change in DLCO (diffusing capacity of the lung for carbon monoxide)
Time frame: 24 Months
Extent of fibrosis on pulmonary CT (Computed Tomography)
Time frame: From 3 months to 1 month before CAR-T infusion
Extent of fibrosis on pulmonary CT(Computed Tomography)
Time frame: 3 Months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Christian Jorgensen, PUPH principal investigator
University Hospital, Montpellier
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CD19 CAR T Arm in people with Scleroderma, Systemic, run by University Hospital, Montpellier at 4 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Charlotte KAAN (04 67 33 48 53 ext. 33, c-kaan@chu-montpellier.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
modified Rodnan skin score (mRSS) (time frame: 6 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.