CD19-Directed Chimeric Antigen Receptor Autologous T Cells (CART19) for Adolescents and Young Adults With Systemic Lupus Erythematosus (SLE)
Children's Hospital of Philadelphia
Summary
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.
Description
Lupus disease activity is associated with increased numbers of activated naïve B cells and polyclonal expansion of antibody secreting cells, indicating a central role for B cells in the pathogenesis of SLE. While traditional anti-CD19 antibody therapies have been utilized with varying success in the treatment of Systemic lupus erythematosus (SLE), CD19 directed cellular therapies have emerged as an attractive therapeutic option that may lead to immunosuppression-free remission in this population given the ability of CD19 directed CAR T cells to more deeply deplete the B cell compartment. Previ…
Eligibility
- Age range
- 12–29 years
- Sex
- All
- Healthy volunteers
- No
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 12-29 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…
Interventions
- BiologicalCART19
CART19 cells transduced with a lentiviral vector to express anti-CD19 scFv:41-BB:TCRζ, administered by IV injection.
Location
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania