CD19-Directed Chimeric Antigen Receptor Autologous T Cells (CART19) for Lupus
CD19-Directed Chimeric Antigen Receptor Autologous T Cells (CART19) for Adolescents and Young Adults With Systemic Lupus Erythematosus (SLE)
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 10 Years to 29 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Procedures mentioned: Injections
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "During this trial the test article will be CART19 cells transduced with a lentiviral vector to express anti-CD19 scFv:41-BB:TCRζ, administered by IV injection."
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests the safety of CART19 and how well it works for lupus that has not improved with treatment. CART19 uses a patient's own T cells, a type of immune cell, changed to target CD19. The study is for people ages 10–29 with lupus, with or without kidney disease.
This is a single-center, single-arm, open-label phase 1/2 study of CART19 in children and young adults with refractory Systemic lupus erythematosus (SLE), including both patients diagnosed with lupus nephritis (LN) and patients with non-renal Systemic lupus erythematosus (SLE). Phase 1 will evaluate the safety of CART19 in 6-12 patients with Systemic lupus erythematosus (SLE). There is no planned dose escalation, but a dose de-escalation will be made based on the incidence of Dose Limiting Toxicities. Phase 2 will evaluate the efficacy and further evaluate the safety of CART19 in this population.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Sign a consent form before any study procedures.
- Be 10–29 years old.
- Meet the study's rules for a lupus diagnosis and have a positive ANA and/or double-stranded DNA blood test.
- Have active lupus despite treatment. For kidney lupus, a kidney sample taken within the past year must show certain types of disease, and repeated urine tests must meet protein rules. For lupus without kidney disease, meet disease activity scores or be unable to lower prednisone enough because lupus is still active.
- Have had at least 3 months total of standard treatment to lower immune system activity, plus at least one other qualifying lupus treatment.
- Have enough kidney function, with an eGFR score of at least 30, and not be on dialysis.
- Meet the study's liver, heart, and lung function requirements.
- Agree to use accepted birth control if able to have children.
Who can't join
- Have an active, untreated infection, HIV, active hepatitis B, or active hepatitis C.
- Have severe lupus effects on the brain, nerves, or mental health, or known lupus caused by a change in a single gene.
- Have had a stem cell transplant or kidney transplant.
- Have a history of seizures or take medicine to prevent seizures.
- Have received an experimental drug in another study too recently. The waiting time depends on how long the drug stays in the body.
- Cannot or will not stop medicines that lower immune system activity when receiving CART19, or have another health problem that could make treatment unsafe.
- Are pregnant or want to keep breastfeeding. Anyone who could become pregnant must have a negative pregnancy test.
- Will not agree to long-term follow-up.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 1. Signed informed consent form must be obtained prior to any study procedure. Labs or other procedures obtained during routine clinical care may be used for eligibility if obtained within the protocol required window. 2. Patient age must be 10-29 years, inclusive, at time of enrollment. 3. Meeting ACR/EULAR Classification Criteria for SLE 4. ANA positive \> 1:80 and/or double-stranded DNA (dsDNA) positive 5. Active (refractory) disease, defined as follows: a. Lupus nephritis subjects must meet both the following criteria: i. ISN/RPS active nephritis Class III/IV +/- V lupus nephritis diagnosed by biopsy within past 12 months. ii. Persistent and clinically significant: ≥2 measurements with urine protein with either of the following: 1. \> 1mg/mg creatinine 2. \> 0.5 mg/mg creatinine associated with renal dysfunction or low albumin. 3. \> 0.5 mg/mg creatinine in a patient with rising proteinuria after prior complete renal response b. Non-renal SLE subjects must meet either of the following criteria: i. SLEDAI-2K ≥ 8 and clinical SLEDAI-2K ≥ 6 ii. Inability to decrease prednisone ≤7.5mg/day or 0.15mg/kg/day, whichever is lower, due to active disease. 6\. Patients must have had at least 3 months of cumulative conventional therapy defined as: 1. Conventional induction immunosuppressive agent(s) (e.g., mycophenolate mofetil, cyclophosphamide), and 2. At least one additional therapy: i. B-cell directed biologic therapy (e.g., rituximab, belimumab, ofatumumab, obinutuzumab) ii. Calcineurin inhibitor (e.g., tacrolimus, cyclosporine, voclosporin) iii. Other immunosuppressive medication for SLE (e.g., anifrolumab, abatacept, JAK inhibitor) 7. Adequate organ function status 1. Renal: eGFR must be ≥30 and subject cannot be receiving dialysis. 2. Hepatic: Transaminases \< 5x upper limit of normal and serum conjugated (Direct) bilirubin \<1.5x upper limit of normal unless attributable to SLE. If attributable to autoimmune disease, Child-Pugh score must be class A or class B. Child-Pugh score cannot be class C. 3. Cardiac: Shortening fraction \> 28%, left ventricular ejection fraction \>45%, and no evidence of severe pulmonary hypertension 4. Pulmonary: Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and \<Grade 3 hypoxia; DLCO ≥40% (corrected for anemia and/or VA volume if necessary) if PFTs are clinically appropriate as determined by the treating investigator. 8\. Subjects of reproductive potential must agree to use acceptable birth control methods. Exclusion Criteria: 1. Active, untreated infections 2. HIV infection 3. Active Hepatitis B a. Patients must have a negative hepatitis B surface antigen to be enrolled on this study. 4. Active Hepatitis C 5. Patients with severe neuropsychiatric lupus or neurologic manifestations of SLE (e.g. stroke, seizure, psychosis, demyelinating syndromes, organic brain syndrome, or lupus related headaches) 6. Monogenic lupus (known) 7. Previous autologous or allogenic stem cell transplant 8. Previous kidney transplant 9. History of seizure disorder 10. Patients who are on anti-epileptic therapy 11. Participation in a clinical trial in which the patient receives an investigational drug within a time period equal or less than 5.5 half-lives of the investigational agent prior to study enrollment. 12. Subjects who are unwilling or unable to discontinue immunosuppressive medications at the times of CART19 infusion will be excluded from the trial 13. Any comorbidity that in the opinion of the investigators would jeopardize the ability of the subject to tolerate therapy. 14. Pregnant patients. All participants of childbearing potential must have negative pregnancy test. 15. Lactating participants who want to continue breastfeeding. 16. Patients who are unwilling to consent to LTFU
Study sites (1)
PA
- Children's Hospital of Philadelphia · Philadelphia, PA 19104Caitlin Elgarten, MD · Study contact · 267-425-7964 · elgartenc@chop.eduMelissa Varghese · Study contact · 845-553-5358 · varghesem@chop.eduCaitlin Elgarten, MD · Principal investigatorMore trials in Philadelphia