ClinicalTrials.gov record NCT06347718
CAR-T Cells in Systemic B Cell Mediated Autoimmune Disease
CAR-T Cells in Systemic B Cell Mediated Autoimmune Disease
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This interventional trial tests a single infusion of autologous anti‑CD19 CAR T cells in adults with active SLE, SSc, DM, or PM. Participants undergo leukapheresis to collect T cells, receive lymphodepleting chemotherapy, then a single intravenous infusion of the expanded CAR T product; the study is open‑label and non‑randomized. The primary focus is to assess safety, including cytokine release syndrome and CAR T–related neurotoxicity.
What it is studying. The study is testing the safety and biological and clinical effects of a single‑dose, autologous CD19‑directed CAR T cell therapy in adults with active systemic lupus erythematosus, systemic sclerosis, dermatomyositis, or polymyositis. It specifically monitors for cytokine release syndrome and neurotoxicity, and measures clinical response by disease‑specific activity indexes, CAR T and B cell levels, autoantibodies, and manufacturing success up to 24 weeks.
What the researchers hope to learn. To assess safety (incidence and severity of cytokine release syndrome and CAR T–associated neurotoxicity) and to measure clinical response at week 24 using disease‑specific activity indexes. Also to measure CAR T cell expansion and B cell levels, changes in disease‑related autoantibodies, success of the product manufacturing process, and physician/patient global assessments and functional questionnaires up to 24 weeks.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The investigational product is designed to effectively combat B cells in patients with autoimmune diseases. Autologous T cells enriched with CD4/CD8 are genetically engineered using a lentiviral vector to express chimeric antigen receptors (CARs) that target the CD19 antigen on the cell surface of B cells and their precursors. During treatment, patients undergo leukapheresis, lymophodepleting chemotherapy and administration of the expanded CD19-CAR-transduced T cells.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- anti-CD19 CAR T cell therapy
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- General:
- Subjects must understand and voluntarily sign an informed consent form including written consent for data protection,
- Adults aged ≥ 18 years at time of consent,
- Adequate renal (eGFR > 30 ml/min/m2), liver (no Child Pugh C), heart (at worst NYHA III, EF > 30%) and pulmonary (FV and DLCO > 30%) function,
- 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 in-dex <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,
- Double vaccination against SARS-CoV-2 or SARS-CoV-2 within the last 6 months.
- SLE specific:
- Fulfilling the 2019 ACR/EULAR classification criteria of SLE,
- Positivity of anti-dsDNA (> 4 U/l), anti-histone (+ or more), anti-nucleosome (+ or more) or anti-Sm antibodies (+ or more),
- 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),
- Insufficient response or intolerance/ contraindication to glucocorticoids and to at least 2 of the following treatments: hydroxychloroquine, mycophenolate mofetil, belimumab, methotrexate, rituximab, cyclophosphamide. Insufficient response is defined as having increased disease activity based on the definition explained in the previous bullet point.
- SSc specific:
- Fulfilling the 2013 ACR/EULAR classification criteria of SSc),
- Positivity (+ or more) for at least one SSc-specific parameter (Scl70, RNA polymerase, Th/To, RP11/12, U3RNP autoantibodies),
- 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 G/L), (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 or intolerance/ contraindication to at least 2 of the following treatments: mycophenolate mofetil, azathioprine, nintedanib, methotrexate, rituximab. Insufficient response is defined as having increased disease activity based on the definition explained in the previous bullet point.
- DM/PM specific:
- Fulfilling the 2017 ACR/EULAR classification criteria for probable or definite DM or PM,
- Presence of active myositis in muscle biopsy or muscle MRI and/or signs of interstitial lung disease related to DM/PM,
- Positivity (+ or more) for at least one myositis-specific antibody (aminoacyl tRNA synthetases, Mi2, MDA5, SAE, SRP, ARS, HMGCR, MJ, TIF1gamma),
- Muscle weakness as define by MMT < 142 and 2 of the following criteria: VAS patients Global ≥2cm, VAS physician Global ≥ 2cm, HAQ > 0.25, at least one muscle enzyme > 1.3 times upper limit of normal, VAS global extra muscular activity ≥ 2cm,
- Insufficient response or intolerance/ contraindication to glucocorticoids and to at least 2 of the following treatments: mycophenolate mofetil, ciclosporin A, tacrolimus, methotrexate, rituximab, intravenous immunoglobulins. Insufficient response is defined as having increased disease activity based on the definition explained in the previous bullet point.
Exclusion criteria
- 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 < 8g/dl, absolute CD3+ T cell count < 100/μl,
- Uncontrolled severe concomitant disease, such as cancer (except basal or squamous cell skin cancer) and diabetes mellitus,
- Severely impaired renal (eGFR ≤ 30 ml/min/m2), liver (Child Pugh C), heart (NYHA IV, EF ≤ 30%) and pulmonary (FV and DLCO ≤ 30%) function,
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if the subject were to participate in the study or confounds the ability to interpret data from the study,
- Prior treatment with anti-CD19 antibody therapy, adoptive T cell therapy or any prior gene therapy product (e.g. CAR T cell therapy),
- History of bone marrow/ hematopoietic stem cell or solid organ transplantation,
- Any concomitant severe active infection, e.g. HIV, hepatitis B or C, SARS-CoV 2 (COVID 19), or active tuberculosis as defined by a positive Quantiferon TB-test. If presence of latent tuberculosis is established then treatment according to local guidelines must have been initiated prior to enrollment,
- Diagnosis of severe neuropsychiatric SLE, inclusion body myositis or limited SSc,
- Pregnant or lactating females,
- Females who are intending to conceive during the study,
- Known hypersensitivity to any drug components,
- Malignancy in the last 5 years before screening,
- 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 to understand the aim, importance and consequences of the study and to give 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 interpretation of results,
- Subjects who possibly are dependent on the Sponsor, the Principal Investigator or other Investigators (e.g. family members).
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants undergo leukapheresis (collection of their white blood cells), receive lymphodepleting chemotherapy, and then a single intravenous infusion of autologous CD19‑CAR T cells manufactured from their cells. They will have follow-up visits with physical exams, blood tests to measure CAR T cells and B cells, disease assessments and questionnaires, and monitoring for side effects such as cytokine release syndrome and neurotoxicity (assessed especially in the first 4 weeks after infusion), with follow-up continuing to at least 24 weeks. The study includes collection of clinical data, lab and serology measurements, and validated disease activity scores. Exact visit frequency and tests are determined by the study protocol and study team.
How the study is structured. Open‑label, single‑group (no control), non‑randomized interventional study. Participants give blood cells by leukapheresis, receive lymphodepleting chemotherapy, then a single intravenous infusion of the anti‑CD19 CAR T product. Follow‑up visits and blood tests continue through 24 weeks to monitor safety, immune cells, antibodies, and disease activity.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The listed sites are in Germany. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on polymyositis
This trial is also listed on this site under dermatomyositis.
Important dates
- Study start
- Jul 17, 2023
- Primary completion
- May 31, 2026 (estimated)
- Study completion
- May 31, 2026 (estimated)
- Target enrollment
- 24 participants (estimated)
- Last updated on registry
- Sep 30, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Miltenyi Biomedicine GmbH
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Georg Schett, Prof. Dr. med. univ. 📞 +49 9131 85 32093 ✉ georg.schett@uk-erlangen.de
- Daniela Bohr, Dr.med 📞 +49 9131 85 32093 ✉ daniela.bohr@uk-erlangen.de
Participating sites (1)
- Universitätsklinikum ErlangenRecruiting
Erlangen, Bavaria, 91054 · Germany
- Georg Schett, Prof. Dr.
- Daniela Bohr, Dr.
Terms used on this page · explanations by MedicalUpdates.ai
- investigational product
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- informed consent form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- eGFR
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- DLCO
- A breathing test that measures how well the lungs pass oxygen from the air into the blood.
- childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- at screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- contraindication
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- hydroxychloroquine
- A medicine used for some autoimmune conditions. It appears in eligibility criteria when a study requires or excludes its use.
- biopsy
- Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
- MRI
- A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- ANC
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- Prior treatment
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- CAR T
- A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- Sponsor
- The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
- Principal Investigator
- The doctor or scientist responsible for running the study at a site or overall.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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