CD19 CAR-T cells for treatment-refractory autoimmune diseases: the phase 1/2 CASTLE basket trial.
Fabian Müller, Melanie Hagen, Andreas Wirsching...
https://pubmed.ncbi.nlm.nih.gov/41501497Actively Recruiting
Phase 1
Phase 2
Age: 18Years +
All Genders
ID06347718
CAR-T Cells in Systemic B Cell Mediated Autoimmune Disease
Led by Miltenyi Biomedicine GmbH · Updated on 2025-09-30
24
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating an advanced therapy using genetically engineered T cells to target B cells in patients with systemic autoimmune diseases such as Systemic Lupus Erythematosus (SLE), Systemic Sclerosis (SSc), Dermatomyositis, and Polymyositis. This open-label, non-randomized study evaluates the safety and effects of a single dose of anti-CD19 CAR T cell therapy, designed to combat B cell activity driving these conditions. The study is sponsored by Miltenyi Biomedicine GmbH and includes adults with active disease who have not responded adequately to other treatments. Participants receive a single intravenous infusion of the investigational product, which consists of their own T cells genetically modified to express chimeric antigen receptors targeting CD19 on B cells. Before infusion, patients undergo leukapheresis to collect T cells and receive lymphodepleting chemotherapy to prepare the body. There is no control group; all participants receive the active treatment. The study monitors patients for 24 weeks post-infusion to assess clinical and immune responses. During the study, participants will have scheduled visits for safety assessments, laboratory tests, disease activity evaluations, and quality of life questionnaires up to 24 weeks after treatment. Researchers will measure safety outcomes up to 28 days and multiple efficacy and immune response markers throughout the 24-week follow-up. The study involves a thorough evaluation of disease-specific activity, patient and physician assessments, and functional health questionnaires to understand the therapy's impact over time.
CONDITIONS
Brief Title
CAR-T Cells in Systemic B Cell Mediated Autoimmune Disease
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Subjects must understand and voluntarily sign an informed consent form including written consent for data protection
- Adults aged 18 years or older at time of consent
- Adequate kidney function (eGFR > 30 ml/min/m2)
- Adequate liver function (no Child Pugh C)
- Adequate heart function (at worst NYHA III, EF > 30%)
- Adequate lung function (FV and DLCO > 30%)
- Male subjects must be surgically sterile or agree to use two contraceptive methods during the trial and for 12 months after dosing
- Females of childbearing potential must have a negative pregnancy test at screening and agree to use highly effective contraception during the trial and for 12 months after dosing
- Must be able to follow the study visit schedule and protocol requirements
- Double vaccination against SARS-CoV-2 or SARS-CoV-2 infection within the last 6 months
- For SLE: must meet 2019 ACR/EULAR criteria, be positive for specific antibodies, have active disease, and insufficient response or intolerance to specified treatments
- For SSc: must meet 2013 ACR/EULAR criteria, be positive for SSc-specific antibodies, show signs of fast progression, and insufficient response or intolerance to specified treatments
- For DM/PM: must meet 2017 ACR/EULAR criteria, show active myositis or lung disease, be positive for myositis-specific antibodies, have muscle weakness and other activity signs, and insufficient response or intolerance to specified treatments
You will not qualify if you...
- Suitable for less burdensome or approved therapies as judged by investigator
- Low blood counts: ANC < 1000/mm3, ALC < 500/mm3, hemoglobin < 8g/dl, CD3+ T cell count < 100/μl
- Uncontrolled severe diseases such as cancer (except certain skin cancers) or diabetes
- Severely impaired kidney, liver, heart, or lung function
- Any condition posing unacceptable risk or confounding data interpretation
- Prior anti-CD19 antibody, adoptive T cell, or gene therapy including CAR T cell therapy
- History of bone marrow, stem cell, or organ transplantation
- Active severe infections including HIV, hepatitis B or C, COVID-19, or active tuberculosis
- Diagnosed severe neuropsychiatric SLE, inclusion body myositis, or limited SSc
- Pregnant or breastfeeding females
- Females planning to conceive during the study
- Known allergy to any study drug components
- Cancer within the last 5 years
- Need for live vaccines during the study or 14 days before leukapheresis
- Under 18 years old or unable to give informed consent
- Recent history of alcohol or substance abuse affecting safety or results
- Possible dependence on study staff or investigators
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Single-dose treatment with assessment up to 28 days
Participants receive a single intravenous infusion of anti-CD19 CAR T cell therapy after leukapheresis and lymphodepleting conditioning.
1 infusion visit and multiple follow-up visits up to day 28
Follow-up
Duration - Up to 24 weeks
Participants are monitored for clinical efficacy, cellular and serological responses, and quality of life up to 24 weeks after treatment.
Regular visits for assessments up to week 24
Trial Site Locations
Total: 1 location
1
Universitätsklinikum Erlangen
Erlangen, Bavaria, Germany, 91054
Actively Recruiting
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Research Team
G
Georg Schett, Prof. Dr. med. univ.
D
Daniela Bohr, Dr.med
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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