Ireland
--:--IST
Active, not recruiting Phase 2

Phase IIa Trial of Anti-CD19 CAR T-Cells in Systemic Sclerosis Resistant to Immunosuppressive Therapy

NCT07493395 · tracked via the Priya Life Science France tracker
Phase
Phase 2
Started
2026-06-11
Last updated
2026-09-16

Condition(s) studied

Scleroderma, Systemic

Investigational drug(s) / intervention(s)

CD19 CAR T Arm

CD19 CAR T Arm: 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 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.

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
64 Years
Healthy volunteers
No
Pre-Inclusion criteria: 1. Diagnosis of systemic sclerosis according to ACR/EULAR 2013 classification (15).we include in the critera the fulfilling of 2013 EULAR/ACR criteria and specify disease duration (less than 2 years), score/clinical evidence for active disease : 2. Severe and resistant to low dose steroids and at least 2 immunosuppressive treatment including csDMARDs (methotrexate, azathioprine, mycophenolate mofetil) and at least one bDMARDs (Tocilizumab) 3. Early onset (less than 2 years). 4. Severity \& progression of disease be defined by : 1. .mRSS \>15 with at least one organ involvement (lung: FVC \<80%, renal involvement, cardiac involvement, Creatinine \< 1.5 mg/dl within 6 months). 2. mRSS \<15 and lung fibrosis progression (FVC -10% DLCO -15% within 6 months) 5. patients with active disease (as defined by EUSTAR ≥2.5) and to patients with a worsening disease despite 6 months of at least 2 immunosuppressive treatments including one DMARDs (methotrexate, azathioprine, mycophenolate mofetil), and one biological DMARD rituximab or tocilizumab. 6. Estimated survival time \> 24 weeks 7. Age: ≥18 ≤64 years old voluntary to participate in the study and sign the informed consent 8. Adequate organ functions assessed : 1. serum Creatinine clearance \> 40ml/mi 2. adequate bone marrow function (Hemoglobin ≥9g/dL ; PMN ≥ 1 G/L ; Platelets ≥ 100 G/L) 3. 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) 4. 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 9. Highly effective contraception methods Inclusion criteria: 1. Adequate organ functions assessed: 1. serum Creatinine clearance \> 40ml/mi 2. adequate bone marrow function (Hemoglobin ≥9g/dL ; PMN ≥ 1 G/L ; Platelets ≥ 100 G/L) 3. 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) 4. 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 2. Adequate venous access for apheresis 3. Leucapheresis : a wash-out period of 6 weeks for conventional immunosuppressants (i.e. methotrexate, mycophenolate mofetil) 4. Leucapheresis : at least 12 weeks after biotherapy (i.e. tocilizumab, 6 months for rituximab), Exclusion Criteria: 1. Craniocerebral trauma, conscious disturbance, epilepsy, cerebrovascular ischemia or cerebrovascular hemorrhagic diseases 2. ECG showing prolonged QT interval or history of severe heart diseases or FEVG \< 40% 3. Lung and / or heart severe dysfunction defined by CVF\<50% and/or DLCO \<40% 4. Pulmonary arterial hypertension defined by catheterism (mean AP \> 25mmHg at rest or \> 30mmHg after exercise, PAOP \< 15mmHG) 5. 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 6. Contra indication for autologous hematopoietic stem cell transplantation (AHSCT ) or relapsing at least one year after AHSCT 7. Active hematological or solid neoplasm 8. Concurrent therapy with systemic steroids (\>10 mg/d prednisone equivalent) within 2 weeks prior to inclusion, except inhaled steroids 9. Methylprednisolone or prednisone (maximum dose 20 mg) instead of immunosuppressive agents 10. T cell targeting drugs (e.g. mycophenolate mofetil, azathioprine, calcineurin inhibitors) within 6 weeks prior to leukapheresis 11. Previous adoptive T cell therapy or any gene therapy including CAR T cell therapy 12. Live vaccines within 6 weeks prior to leukapheresis 13. 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 14. patients without social security coverage; 15. patients under guardianship; 16. Male or female patients seeking to conceive a child 17. 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. 18. pregnant or breastfeeding women; 19. patients with advanced cognitive disorders or any other cause preventing their informed consent; 20. 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 21. 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. 22. Concurrent participation in any other interventional trial and Contraindication to the lymphodepleting chemotherapy

Primary outcome measure(s)

  • modified Rodnan skin score (mRSS) — 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)

Trial sites (4)

FacilityCityRegionStatus
CHRU Lille Lille France
Montpellier University Hospital Montpellier France
APHP Necker Paris France
Chu Rouen Rouen France
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07493395 on ClinicalTrials.gov ↗ ← All trials in France