Lupus Erythematosus, SystemicPhase 1/2CATARSISNCT06685042
Anti-CD19 CAR T-Cell Therapy in Refractory Systemic Autoimmune Diseases
A Phase 1/2 study testing CAR T cell in people with Lupus Erythematosus, Systemic and System; Sclerosis, run by Fondazione Policlinico Universitario Agostino Gemelli IRCCS at 1 site in Italy.
Trial summary
The CATARSIS study explores the use of anti-CD19 CAR T-cell therapy as a novel approach for treating refractory systemic autoimmune diseases, specifically SLE, SSc, DM/PM, and AAV. These life-threatening conditions often resist current therapies, and B cells play a key role in their pathogenesis. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Lupus Erythematosus, Systemic, System; Sclerosis, ANCA Associated Vasculitis
- What is being tested?
- CAR T cell
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteItaly
- Study dates
- 29 Nov 2024to Dec 2025
- Phase
- Phase 1/2
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- General
- Subjects must understand and voluntarily sign an informed consent form, including written consent for data protection;
- Adults aged ≥ 18 years and < 65 years at time of consent;
- Male subjects, unless surgically sterile, must agree to use two acceptable methods for contraception (e.g., spermicide and condom) during the trial and refrain from fathering a child starting from the time of signing the Informed Consent Form (ICF) until 12 months after dosing of the IMP;
- Females of childbearing potential (FCBP) must have a negative urine pregnancy test at screening and must agree to use a highly effective contraceptive method (Pearl index <1) starting from the time of signing the ICF and for 12 months after dosing of the IMP;
- Must be able to adhere to the study visit schedule and other protocol requirements;
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- Double vaccination (2 doses) against SARS-CoV-2 or SARS-CoV-2 within the last 6 months.
- SLE subjects
- Fulfilling the 2019 ACR/EULAR classification criteria of SLE; b) Presence of anti-dsDNA, anti-histone, anti-nucleosome or anti-Sm antibodies; c) Active disease at screening, defined as ≥1 organ system with a British Isles Lupus Assessment (BILAG) A score (severe disease activity) or ≥2 organ systems with a BILAG B score (moderate disease activity); d) Insufficient response to glucocorticoids and at least 2 of the following treatments: hydroxychloroquine, mycophenolate mofetil, belimumab, methotrexate, rituximab.
- SSc subjects
- Fulfilling the 2013 ACR/EULAR classification criteria of SSc;
- Diffuse SSc with respective autoantibody profile;
- Signs for fast progression including i) disease duration ≤5 years (from onset of first non-Raynaud manifestation), ii) mRSS score 10-35 at screening, iii) elevated acute phase reactant levels (CRP ≥ 6 mg/L, ESR ≥ 28mm/h or platelet count ≥ 330 000/mm3), iii) mRSS increase ≥ 3 units or involvement of one new body area or mRSS increase ≥ 2 units in one body area or ≥1 tendon friction rub over 6 months;
- Insufficient response to glucocorticoids and to at least 2 of the following treatments:
- mycophenolate mofetil, azathioprine, nintedanib, methotrexate, rituximab.
- DM/PM subjects
- Fulfilling the 2017 ACR/EULAR classification criteria for probable or definite DM or PM57, b) Muscle weakness as defined by MMT < 142 and 2 of the following criteria: VAS patients Global ≥ 2 cm, VAS physician Global ≥ 2 cm, HAQ > 0.25, at least one muscle enzyme >1.3 times upper limit of normal, VAS global extra muscular activity ≥ 2 cm; c) Presence of at least one myositis-specific antibody; d) Insufficient response to glucocorticoids and to at least 2 of the following treatments: mycophenolate mofetil, ciclosporin A, tacrolimus, methotrexate, rituximab, and intravenous immunoglobulins.
- AAV subjects
- Fulfilling the 2022 ACR/EULAR classification criteria for MPA/GPA/EGPA,
- Presence of ANCA to either proteinase 3 or myeloperoxidase;
- At least one major or three non-major items or at least two renal items of hematuria and proteinuria on the BVAS;
- Failure of at least 1 of the following treatments: glucocorticoids, cyclophosphamide, or B-cell targeting therapy.
Who cannot join
- Clinically suitability for a less burdensome and/or approved therapeutic approach, as judged by the investigator;
- ANC < 1.000/mm3, ALC < 500/mm3 or hemoglobin < 8 g/dl, absolute CD3+ T cell count ≤100/µl;
- Evidence of significant and uncontrolled concomitant diseases that could affect compliance with the protocol or interpretation of results.
- Relevant cardiovascular disease: recent history of myocardial infarction, cardiac angioplasty or stenting, unstable angina, significant arrhythmia, congestive heart failure, or left ventricular ejection fraction < 50%, as determined by echocardiography
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study;
- Impaired renal function, i.e., eGFR < 30 ml/min;
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- Patients with evidence on thorax CT of advanced fibrotic interstitial lung disease and whose latest pulmonary function test showed a Forced Vital Capacity (FVC) < 40% of predicted or a Diffusing Capacity for Carbon Monoxide (DLCO) < 30% of predicted
- Any concomitant severe active infection, including HIV (even with negative viral load), active hepatitis B (either positive for Hepatitis B core antibody [HBcAb] or positive hepatitis B surface antigen [HBsAg] and NAT tests) and/or C (<12 weeks between achievement of a sustained virological response to the specific treatment and apheresis) according to the American Association for the Study of Liver Diseases guidelines, SARS-CoV 2 (COVID 19), or active tuberculosis as defined by a positive Quantiferon TB-test. If the presence of latent tuberculosis is established, then treatment according to local guidelines must have been initiated before enrolment;
- Pregnant or lactating females;
- Known hypersensitivity to either any drug components or any auxiliary medicinal products scheduled during trial participation, including during lymphodepletion;
- Malignancy in the last 5 years before screening. The inclusion of patients with previously completely resected carcinoma in situ who have not required treatment other than surgery is allowed.
- Previous CAR T cell administration;
- A therapeutic schedule not compatible with the wash-out requirements for the leukapheresis procedure (section 5.8.1 of CSP) and the medications permitted during the study (section 7.11 of CSP);
- Concurrent treatment with other investigational agents or participation in other investigational trials.
- Treatment, as part of an investigational clinical trial, with an experimental product with a definite or potential effect on T or B-cells in the previous 2 years.
- Requirement for immunization with live vaccine during the study period or within 14 days preceding leukapheresis;
- Subjects who are younger than 18 years or are incapable of understanding the aim, importance, and consequences of the study and giving legal informed consent;
- Have a history of alcohol or substance abuse within the preceding 6 months that, in the opinion of the Investigator, may increase the risks associated with study participation or study agent administration or may interfere with the interpretation of results;
- Subjects who possibly are dependent on the Sponsor, the Principal Investigator, or the Investigator (e.g., family members).
- Limited to patients diagnosed with SLE: patients with a history of severe central nervous system (CNS) involvement, including those who have presented aseptic meningitis, cerebral vasculitis, cerebrovascular disease, demyelinating syndrome, myelopathy, seizure disorder, status epilepticus, and severe lupus headache, will be excluded.
- Patients meeting the classification criteria for multiple connective diseases such as overlapping SLE and Sjogren's Syndrome (SS) or SLE and Rheumatoid Arthritis (RA) and patients diagnosed with Mixed Connective Tissue Disease (MCTD).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site, 1 recruiting
- Recruiting
Fondazione Policlinico Universitario A. Gemelli IRCCS
Rome, Lazio, 00168
Giulia Wlderk, 0630159650, giulia.wlderk@policlinicogemelli.it
About this study
The CATARSIS study explores the use of anti-CD19 CAR T-cell therapy as a novel approach for treating refractory systemic autoimmune diseases, specifically SLE, SSc, DM/PM, and AAV. These life-threatening conditions often resist current therapies, and B cells play a key role in their pathogenesis. The study employs CD19-CAR_Lenti, an autologous CAR T-cell product targeting CD19-positive B cells, aiming to reduce inflammation and autoimmunity. This open-label, single-dose, phase I basket trial will assess the safety, feasibility, and preliminary efficacy of CAR T-cell therapy, focusing on adverse events, infection rates, and overall response at 24 weeks. Eight participants will be included.
Read the detailed description
In recent years, B cells have become more appreciated as major players and also as therapeutic targets in systemic autoimmune diseases. Systemic autoimmune diseases, such as Systemic Lupus Erythematosus (SLE), Systemic Sclerosis (SSc), Dermatomyositis/Polymyositis (DM/PM), and ANCA Associated Vasculitis (AAV) are among the most life-threatening diseases. Even though clinical presentation and organ involvement may be different, these diseases share common pathways of B cell activation. Despite the substantial advances in their management, some patients with systemic autoimmune diseases fail to respond to the current state-of-the art therapies, and are at high risk for severe organ failure and even death. Therefore, new treatments for such refractory cases are urgently needed.
A strong preclinical rationale supports the use of anti-CD19 CAR T cells in systemic autoimmune disease. Experimental animal models back the concept that anti-CD19 CAR T cell therapy could be a powerful approach to target autoimmunity and inflammation in B cell mediated autoimmune disease. "CD19-CAR_Lenti" (the ATMP) consists of autologous CD19 Chimeric Antigen Receptor (CAR) transduced CD4/CD8 enriched T cells, derived from a leukapheresis product and processed at Officina Farmaceutica of IRCCS Ospedale Pediatrico Bambino Gesù through a validated process by using the CliniMACS Prodigy® device. CD19-CAR_Lenti is a suspension of fresh CD4/CD8- enriched CD3+ T cells that have been gene-modified with a self-inactivating (SIN) lentiviral vector expressing a CAR directed against human CD19.
CATARSIS is a basket study on four prototypic systemic autoimmune diseases, which a) show a severe life-threatening disease course, b) are associated with a current high unmet need for treatment, c) show an involvement of B lymphocytes in disease pathogenesis and d) respond at least partially to treatment with B cell depleting antibodies.
The investigators will conduct a prospective, open-label, single-dose, non-randomized, interventional phase I clinical study, using a "basket" design, to assess the safety, feasibility, and preliminary efficacy of ex vivo generated autologous anti-CD19 CAR T cells targeting a common pathogenic pathway (activated B cells and plasmablasts) in subjects with severe, treatment-failure systemic autoimmune disease (SLE, SSc, DM/PM and AAV).
In particular, the investigators will assess incidence and grading of severity of Cytokine Release Syndrome (CRS) and of CAR T cell Associated Neurotoxicity Syndrome (ICANS) within the first 4 weeks after ATMP administration, incidence and severity of infections and leukopenia and/or hypogammaglobulinemia during the entire study period (24 weeks), and overall Response Rate (ORR) at week 24 based on specific disease activity composite indexes, each of them validated for the specific disease.
Duration of B cell depletion in the peripheral blood, duration of persistence of CAR T cells in the peripheral blood, and changes in the levels of disease-associated serum autoantibodies will be assessed as well through specific laboratory measurements.
A total of 8 subjects with B cell-mediated autoimmune disease will be included in the CATARSIS study, following a Bryant and Day two stages design. Subjects will receive a single intravenous infusion of 1 x 10^6 /kg body weight CAR T cell. Recruitment and outpatient visits will be performed in the UOC Reumatologia - Dipartimento di scienze dell'Invecchiamento, Ortopediche e Reumatologiche (FPG) and Dipartimento Oncoematologia, Terapia Cellulare, Terapie Geniche e Trapianto Emopoietico (OPBG). Treatment and pre-treatment specific procedures (leukapheresis, lymphodepletion) of subjects and the inpatient visits will be performed in the UOC Ematologia e Trapianto di cellule staminali emopoietiche - Dipartimento di diagnostica per Immagini, Radioterapia Oncologica ed Ematologia (FPG) and Dipartimento Oncoematologia, Terapia Cellulare, Terapie Geniche e Trapianto Emopoietico (OPBG) by trained investigators. ATMP manufacturing will be performed in a certified facility at Officina Farmaceutica, IRCCS Ospedale Pediatrico Bambino Gesù, under licenses by local regulatory authorities and highest technical standards.
- Conditions
- Lupus Erythematosus, Systemic, System; Sclerosis, ANCA Associated Vasculitis, Dermatomyositis, Polymyositis
- Being tested
- CAR T cell
- Medical subject headings
- Lupus Erythematosus, Systemic, Scleroderma, Diffuse, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, Dermatomyositis, Polymyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CAR T cellBiological
The investigational medicinal product (IMP) "CD19-CAR_Lenti" consists of autologous CD19 Chimeric Antigen Receptor (CAR) transduced CD4/CD8 enriched T cells, derived from a leukapheresis product and processed by using the CliniMACS Prodigy® device (Miltenyi Biotec). CD19-CAR_Lenti is a suspension of fresh CD4/CD8-enriched CD3+ T cells that have been genemodified with a self-inactivating (SIN) lentiviral vector expressing a CAR directed against human CD19. The SIN lentiviral vector is derived from clinically validated viral vectors described in the literature with a fully characterized sequence.
| Study group | Role | What they receive |
|---|---|---|
| Anti-CD19 CAR T cell | Experimental | Single intravenous infusion of 1 x 106 /kg body weight CAR T cell ("CD19-CAR_Lenti" ) Receives: Biological: CAR T cell |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects experiencing a CRS or an ICANS
The first safety outcome variable will be measured as the number of subjects experiencing a Cytokine Release Syndrome (CRS) or an (Immune Effector Cell-associated Neurotoxicity Syndrome) ICANS within the first 4 weeks after ATMP administration in relation to the number of subjects enrolled in the study.
Measured: 4 weeks
Incidence of infections, leukopenia and/or hypogammaglobulinemia
The second safety outcome variable will be measured as the incidence of infections, leukopenia, and/or hypogammaglobulinemia during the entire study period in relation to the number of subjects enrolled in the study.
Measured: 24 weeks
Overall Response Rate
The efficacy outcome variable, Overall Response Rate (ORR), is defined as the ratio between the number of subjects experiencing a response at week 24 and the total number of enrolled subjects.
A response to treatment will be considered at week 24 as:
- SLE: Fulfillment of DORIS remission criteria of SLE.
- SSc: No progression of interstitial lung disease with worsening of FVC (>10%) or worsening of FVC (5-10%) plus an increase in respiratory symptoms or worsening of FVC (5-10%) plus progression of high-resolution computed tomography changes after 24 weeks.
- DM/PM: 2016 ACR/EULAR Moderate or Major Response.
- AAV: Birmingham vasculitis activity score (BVAS) of 0.
Measured: 24 weeks
Secondary outcomes (3)
Time of persistence of CAR T cells
Time of persistence of CAR T cells in the peripheral blood is defined as the time elapsed from the date of the study intervention and the date of disappearance of CAR T cells. CAR T cells will be identified as proportion of CAR+ cells relative to total CD3+ cells (0-100%) and in absolute number (cells per milliliter of peripheral blood).
Measured: 24 weeks
Time of absence of B cells in the peripheral blood
Time of absence of B cells in the peripheral blood is defined as the time elapsed from the date of the study intervention and the date of the rise of B cells after the intervention-induced nadir. B cells will be identified as proportion of CD19+ cells relative to total lymphocytes (0-100%) and in absolute number (cells per milliliter of peripheral blood).
Measured: 24 weeks
Time to the disappearance of autoantibodies in the serum
Time to the disappearance of autoantibodies in the serum is defined as the time elapsed from the date of the study intervention and the date of negativization of disease-specific autoantibodies. The negativity of an autoantibody is defined as its detection at a concentration (AU/mL) below the lower limit of positivity, according to the laboratory's reference ranges.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Nov 2024
- Study start29 Nov 2024
- Primary completionDec 2025 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lupus Erythematosus, SystemicStudies of the same condition, open and under way.
- More trials run by Fondazione Policlinico Universitario Agostino Gemelli IRCCSThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, D'Agostino Maria Antonietta, Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Investigators
- Maria Antonietta D'Agostino, Principal investigator, Fondazione Policlinico Universitario Agostino Gemelli IRCCS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06685042
- Sponsor's protocol ID
- 6192
- Other IDs
- 2024-511293-74-01
- Acronym
- CATARSIS
- Status last verified
- Apr 2025
- First submitted
- 25 Oct 2024
- Last updated
- 11 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Anti-CD19 CAR T-Cell Therapy in Refractory Systemic Autoimmune Diseases
- ClinicalTrials.gov ID
- NCT06685042
- Acronym / sponsor ID
- CATARSIS / 6192
- Conditions
- Lupus Erythematosus, Systemic, System; Sclerosis, ANCA Associated Vasculitis, Dermatomyositis, Polymyositis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCSUniversity, hospital or other
- Study start
- 29 Nov 2024 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 12 Nov 2024 (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 CATARSIS study explores the use of anti-CD19 CAR T-cell therapy as a novel approach for treating refractory systemic autoimmune diseases, specifically SLE, SSc, DM/PM, and AAV. These life-threatening conditions often resist current therapies, and B cells play a key role in their pathogenesis. The study employs CD19-CAR_Lenti, an autologous CAR T-cell product targeting CD19-positive B cells, aiming to reduce inflammation and autoimmunity. This open-label, single-dose, phase I basket trial will assess the safety, feasibility, and preliminary efficacy of CAR T-cell therapy, focusing on adverse events, infection rates, and overall response at 24 weeks. Eight participants will be included.
Detailed description
In recent years, B cells have become more appreciated as major players and also as therapeutic targets in systemic autoimmune diseases. Systemic autoimmune diseases, such as Systemic Lupus Erythematosus (SLE), Systemic Sclerosis (SSc), Dermatomyositis/Polymyositis (DM/PM), and ANCA Associated Vasculitis (AAV) are among the most life-threatening diseases. Even though clinical presentation and organ involvement may be different, these diseases share common pathways of B cell activation. Despite the substantial advances in their management, some patients with systemic autoimmune diseases fail to respond to the current state-of-the art therapies, and are at high risk for severe organ failure and even death. Therefore, new treatments for such refractory cases are urgently needed.
A strong preclinical rationale supports the use of anti-CD19 CAR T cells in systemic autoimmune disease. Experimental animal models back the concept that anti-CD19 CAR T cell therapy could be a powerful approach to target autoimmunity and inflammation in B cell mediated autoimmune disease. "CD19-CAR_Lenti" (the ATMP) consists of autologous CD19 Chimeric Antigen Receptor (CAR) transduced CD4/CD8 enriched T cells, derived from a leukapheresis product and processed at Officina Farmaceutica of IRCCS Ospedale Pediatrico Bambino Gesù through a validated process by using the CliniMACS Prodigy® device. CD19-CAR_Lenti is a suspension of fresh CD4/CD8- enriched CD3+ T cells that have been gene-modified with a self-inactivating (SIN) lentiviral vector expressing a CAR directed against human CD19.
CATARSIS is a basket study on four prototypic systemic autoimmune diseases, which a) show a severe life-threatening disease course, b) are associated with a current high unmet need for treatment, c) show an involvement of B lymphocytes in disease pathogenesis and d) respond at least partially to treatment with B cell depleting antibodies.
The investigators will conduct a prospective, open-label, single-dose, non-randomized, interventional phase I clinical study, using a "basket" design, to assess the safety, feasibility, and preliminary efficacy of ex vivo generated autologous anti-CD19 CAR T cells targeting a common pathogenic pathway (activated B cells and plasmablasts) in subjects with severe, treatment-failure systemic autoimmune disease (SLE, SSc, DM/PM and AAV).
In particular, the investigators will assess incidence and grading of severity of Cytokine Release Syndrome (CRS) and of CAR T cell Associated Neurotoxicity Syndrome (ICANS) within the first 4 weeks after ATMP administration, incidence and severity of infections and leukopenia and/or hypogammaglobulinemia during the entire study period (24 weeks), and overall Response Rate (ORR) at week 24 based on specific disease activity composite indexes, each of them validated for the specific disease.
Duration of B cell depletion in the peripheral blood, duration of persistence of CAR T cells in the peripheral blood, and changes in the levels of disease-associated serum autoantibodies will be assessed as well through specific laboratory measurements.
A total of 8 subjects with B cell-mediated autoimmune disease will be included in the CATARSIS study, following a Bryant and Day two stages design. Subjects will receive a single intravenous infusion of 1 x 10^6 /kg body weight CAR T cell. Recruitment and outpatient visits will be performed in the UOC Reumatologia - Dipartimento di scienze dell'Invecchiamento, Ortopediche e Reumatologiche (FPG) and Dipartimento Oncoematologia, Terapia Cellulare, Terapie Geniche e Trapianto Emopoietico (OPBG). Treatment and pre-treatment specific procedures (leukapheresis, lymphodepletion) of subjects and the inpatient visits will be performed in the UOC Ematologia e Trapianto di cellule staminali emopoietiche - Dipartimento di diagnostica per Immagini, Radioterapia Oncologica ed Ematologia (FPG) and Dipartimento Oncoematologia, Terapia Cellulare, Terapie Geniche e Trapianto Emopoietico (OPBG) by trained investigators. ATMP manufacturing will be performed in a certified facility at Officina Farmaceutica, IRCCS Ospedale Pediatrico Bambino Gesù, under licenses by local regulatory authorities and highest technical standards.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Anti-CD19 CAR T cell | Experimental | Biological: CAR T cellSingle intravenous infusion of 1 x 106 /kg body weight CAR T cell ("CD19-CAR_Lenti" ) |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- General
- SLE subjects
- SSc subjects
- mycophenolate mofetil, azathioprine, nintedanib, methotrexate, rituximab.
- DM/PM subjects
Key exclusion criteria (21 listed)
- Clinically suitability for a less burdensome and/or approved therapeutic approach, as judged by the investigator;
- ANC < 1.000/mm3, ALC < 500/mm3 or hemoglobin < 8 g/dl, absolute CD3+ T cell count ≤100/µl;
- Evidence of significant and uncontrolled concomitant diseases that could affect compliance with the protocol or interpretation of results.
- Relevant cardiovascular disease: recent history of myocardial infarction, cardiac angioplasty or stenting, unstable angina, significant arrhythmia, congestive heart failure, or left ventricular ejection fraction < 50%, as determined by echocardiography
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study;
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 |
|---|---|---|
| CAR T cellThe investigational medicinal product (IMP) "CD19-CAR_Lenti" consists of autologous CD19 Chimeric Antigen Receptor (CAR) transduced CD4/CD8 enriched T cells, derived from a leukapheresis product and processed by using the CliniMACS Prodigy® device (Miltenyi Biotec). CD19-CAR_Lenti is a suspension of fresh CD4/CD8-enriched CD3+ T cells that have been genemodified with a self-inactivating (SIN) lentiviral vector expressing a CAR directed against human CD19. The SIN lentiviral vector is derived from clinically validated viral vectors described in the literature with a fully characterized sequence. | Biological | Anti-CD19 CAR T cell |
Primary outcome
Number of subjects experiencing a CRS or an ICANS
Time frame: 4 weeks
The first safety outcome variable will be measured as the number of subjects experiencing a Cytokine Release Syndrome (CRS) or an (Immune Effector Cell-associated Neurotoxicity Syndrome) ICANS within the first 4 weeks after ATMP administration in relation to the number of subjects enrolled in the study.
Incidence of infections, leukopenia and/or hypogammaglobulinemia
Time frame: 24 weeks
The second safety outcome variable will be measured as the incidence of infections, leukopenia, and/or hypogammaglobulinemia during the entire study period in relation to the number of subjects enrolled in the study.
Overall Response Rate
Time frame: 24 weeks
The efficacy outcome variable, Overall Response Rate (ORR), is defined as the ratio between the number of subjects experiencing a response at week 24 and the total number of enrolled subjects. A response to treatment will be considered at week 24 as: * SLE: Fulfillment of DORIS remission criteria of SLE. * SSc: No progression of interstitial lung disease with worsening of FVC (>10%) or worsening of FVC (5-10%) plus an increase in respiratory symptoms or worsening of FVC (5-10%) plus progression of high-resolution computed tomography changes after 24 weeks. * DM/PM: 2016 ACR/EULAR Moderate or Major Response. * AAV: Birmingham vasculitis activity score (BVAS) of 0.
3 secondary outcomes
Time of persistence of CAR T cells
Time frame: 24 weeks
Time of absence of B cells in the peripheral blood
Time frame: 24 weeks
Time to the disappearance of autoantibodies in the serum
Time frame: 24 weeks
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 11 Apr 2025.
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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.
Maria Antonietta D'Agostino principal investigator
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CAR T cell in people with Lupus Erythematosus, Systemic and System; Sclerosis, run by Fondazione Policlinico Universitario Agostino Gemelli IRCCS at 1 site in Italy.
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 22 inclusion and 21 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 11 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 Maria Antonietta D'Agostino ((+39)0630159650, mariaantonietta.dagostino@policlinicogemelli.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of subjects experiencing a CRS or an ICANS (time frame: 4 weeks). The study lists 3 primary outcomes in all. 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 11 Apr 2025.