REACT-01: Reversing Autoimmunity Through Cell Therapy

Overview

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

• Biological SCRI-CAR19v3
  Single infusion of SCRI-CAR19v3

Primary outcome measures

• Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [Time frame: 28 days post-infusion]
• Rate of SCRI-CAR19v3 Manufacturing Success [Time frame: 28 days]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

United States · 1 center

• Seattle Children's Hospital — Seattle

Identifiers

NCT: NCT06465147 · REACT-01

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗