Systemic Lupus Erythematosus
Conditions
Keywords
CAR T cells, Lupus, Systemic Lupus Erythematosus, SLE
Brief summary
This is a phase 1, open-label, non-randomized study enrolling pediatric and young adult research participants with treatment-refractory Systemic Lupus Erythematosus (SLE), to examine the safety, feasibility, and efficacy of administering T cell products derived from peripheral blood mononuclear cells (PBMC) that have been genetically modified to express CD19 specific chimeric antigen receptor (CAR) A child or young adult meeting all eligibility criteria and meeting none of the exclusion criteria will have their T cells collected. The T cells will then be bioengineered into a CAR T cell that targets circulating and tissue residing B cells.
Interventions
BIOLOGICALSCRI-CAR19v3
Single infusion of SCRI-CAR19v3
Sponsors
Seattle Children's Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 30 Years
Healthy volunteers
No
Inclusion criteria
* Male and female subjects aged between 2-30 years old. The first 3 subjects will be aged ≥ 17. The FDA will review safety data to determine if the age can be lowered first to ≥ 12 then, following the treatment of 3 further subjects aged 12-17, to ≥ 2 * Serologically active Systemic Lupus Erythematosus that is refractory to treatment * Able to tolerate apheresis or already has an apheresis product available for use in manufacturing. * ≥ 24 weeks post last Rituximab or related B cell depleting therapy * ≥ 12 weeks post last Belimumab / Anifrolumab therapy * ≥ 4 weeks post last calcineurin inhibitor treatment * For subjects receiving non-calcineurin immunosuppressive therapy, on a stable dose for ≥ 8 weeks before enrollment * For subjects receiving corticosteroid therapy, on a stable dose for ≥ 2 weeks before enrollment * Adequate organ function * Adequate laboratory values * Subjects of childbearing or child-fathering potential must agree to use highly effective contraception from consent through 12 months following infusion of investigational product on trial * Subjects must be willing to remain within 1 hour's drive of Seattle Children's Hospital for 4 weeks following CAR T cell infusion. * Subject and/or legally authorized representative has signed the informed consent form for this study
Exclusion criteria
* History or presence of active CNS lupus or other CNS disease * Kidney dysfunction requiring renal replacement therapy * Pregnant or breastfeeding * Insufficient pulmonary reserve including history of COPD, \>10 pack year smoking history or SLE lung disease with hypoxia at rest with oxygen saturation ≤92% on room air * Unable to tolerate repletion with any formulation of IgG. * Active or prior malignancy, unless the malignancy was treated and there is no evidence of recurrent disease \<5 years from enrollment. * Prior solid organ transplantation. * Presence of an active severe infection * Presence of any condition that, in the opinion of the investigator, would prohibit the subject from undergoing treatment under this protocol
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] | 28 days post-infusion | The investigators will assess and described the type, frequency, severity, and duration of adverse events associated with the CAR T cell product. |
| Rate of SCRI-CAR19v3 Manufacturing Success | 28 days | We will measure the number of successfully manufactured SCRI-CAR19v3 products. |
Countries
United States
Contacts
Primary ContactShaun Jackson, MD
Outcome results
None listed