Actively Recruiting
CD19-Directed Chimeric Antigen Receptor Autologous T Cells (CART19) for Adolescents and Young Adults With Systemic Lupus Erythematosus (SLE)
Led by Children's Hospital of Philadelphia · Updated on 2026-02-23
24
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the use of CART19, a CD19-directed chimeric antigen receptor autologous T cell therapy, in children and young adults aged 12 to 29 years with refractory Systemic Lupus Erythematosus (SLE), including those with lupus nephritis (LN) and non-renal SLE. This phase 1/2 open-label study aims to assess the safety and effectiveness of CART19 cells, which are designed to deeply reduce B cells that play a central role in lupus activity. Previous treatments targeting CD19 have had mixed results, and this trial explores a new cellular therapy approach that may lead to remission without ongoing immunosuppression. Participants will receive an intravenous infusion of the study treatment, which consists of CART19 cells engineered via a lentiviral vector to express anti-CD19 scFv:41-BB:TCRζ. The study includes a phase 1 safety evaluation with dose de-escalation if needed, followed by a phase 2 assessment of effectiveness and safety. The treatment is given once, and patients will be monitored for up to 24 months after infusion to assess outcomes and side effects. During the study, participants will undergo regular assessments including laboratory tests, clinical evaluations, and monitoring of CART19 cell behavior such as expansion and persistence in the body. Researchers will measure the frequency of dose-limiting toxicities, rates of clinical remission without steroids, renal response, survival, flare-free survival, cytokine levels, and the feasibility of producing CART19 cells. Safety follow-up and detailed monitoring will occur throughout the two-year period after treatment to understand the long-term effects and impacts of CART19 therapy.
CONDITIONS
Brief Title
CD19-Directed Chimeric Antigen Receptor Autologous T Cells (CART19) for Lupus
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent form obtained before any study procedure
- Age 12 to 29 years inclusive at enrollment
- Meets ACR/EULAR Classification Criteria for SLE
- ANA positive > 1:80 and/or double-stranded DNA positive
- Active refractory disease: for lupus nephritis, ISN/RPS Class III/IV +/- V biopsy within 12 months and persistent significant proteinuria; for non-renal SLE, SLEDAI-2K score ≥ 8 and clinical SLEDAI-2K ≥ 6 or inability to reduce prednisone below specified dose
- At least 3 months of conventional therapy including induction immunosuppressive agent and additional biologic or immunosuppressive therapy
- Adequate organ function: renal (eGFR ≥30, no dialysis), hepatic (transaminases <5x upper limit, bilirubin <1.5x upper limit unless SLE related, Child-Pugh class A or B), cardiac (shortening fraction >28%, ejection fraction >45%, no severe pulmonary hypertension), pulmonary (Grade ≤1 dyspnea, <Grade 3 hypoxia, DLCO ≥40% if tested)
- Subjects of reproductive potential must agree to use acceptable birth control methods
You will not qualify if you...
- Active, untreated infections
- HIV infection
- Active Hepatitis B infection; negative hepatitis B surface antigen required
- Active Hepatitis C infection
- Severe neuropsychiatric lupus or major neurologic SLE manifestations
- Known monogenic lupus
- Previous autologous or allogenic stem cell transplant
- Previous kidney transplant
- History of seizure disorder or current anti-epileptic therapy
- Participation in another investigational drug trial within specified washout period
- Unable or unwilling to discontinue immunosuppressive medications at CART19 infusion
- Any comorbidity that may prevent tolerating therapy
- Pregnant or lactating patients; negative pregnancy test required
- Unwillingness to consent to long-term follow-up monitoring (LTFU)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single treatment session with follow-up
Participants receive an intravenous infusion of CART19 cells, which are genetically modified T cells targeting CD19 in lupus.
1 infusion visit followed by multiple follow-up visits over 24 months
Duration - Up to 24 months post infusion
Participants are monitored for safety, treatment effects, and long-term outcomes including toxicities, disease remission, and survival.
Regular visits over 24 months
Trial Site Locations
Total: 1 location
1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Research Team
C
Caitlin Elgarten, MD
M
Melissa Varghese
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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