This exploratory, single-arm, open-label study will evaluate the safety and preliminary efficacy of YTS109 cell therapy in pediatric patients with relapsed/refractory autoimmune diseases, including systemic lupus erythematosus, diffuse systemic sclerosis, idiopathic inflammatory myopathies, and Sjögren's syndrome, as well as other eligible autoimmune diseases defined by the protocol eligibility criteria. Approximately 12 patients aged 5 to \<18 years will be enrolled at Children's Hospital of Fudan University and will receive a single intravenous infusion of YTS109 cells. Dose escalation will follow a standard 3+3 design starting at 1.5 × 10\^6 cells/kg. The primary objective is to assess the safety and preliminary efficacy of YTS109 cell therapy in this population. Secondary objectives include characterizing the pharmacokinetic and pharmacodynamic profiles of YTS109 cells. Primary endpoints include the type, severity, and frequency of adverse events, along with efficacy assessments.
Age 5 to <18 years at screening; sex not restricted.
CD19 positivity: Presence of CD19-positive B cells in peripheral blood, confirmed by flow cytometry.
Absolute neutrophil count (ANC) ≥ 1.0 × 10^9/L (no colony-stimulating factor use within 2 weeks prior to testing; neutropenia attributable to the underlying disease may be allowed);
Hemoglobin ≥ 60 g/L. 2）Hepatic function: ALT ≤ 3 × ULN (exceptions allowed for elevations attributable to the underlying disease); AST ≤ 3 × ULN (exceptions allowed for elevations attributable to the underlying disease); Total bilirubin (TBIL) ≤ 1.5 × ULN (exceptions allowed for elevations attributable to the underlying disease).
History of severe drug allergy or a known allergic predisposition.
Presence of, or suspected uncontrolled infection requiring treatment, including fungal, bacterial, viral, or other infections.
Central nervous system (CNS) disorders, except for prior seizures, psychosis, organic brain syndrome, cerebrovascular accident, encephalitis, or CNS vasculitis attributable to the underlying disease, as determined by the investigator.
Cardiac dysfunction deemed unable to tolerate study treatment (i.e., inadequate cardiac function at the investigator's discretion).
Single group
YTS109 cell
Biological/VaccineSingle intravenous infusion of YTS109 cells; dose escalation per 3+3 design starting at 0.75×10\^6 cells/kg.
YTS109 cell
Other interventionSingle intravenous infusion of YTS109 cells; dose escalation per 3+3 design starting at 0.75×10\^6 cells/kg.
Incidence of Treatment-Emergent Adverse Events
Safety assessments are conducted using the NCI-CTCAE version 5.0 standards.
Efficacy outcomes for SLE
SLE Response index 4(SR-4) response: Min/Max Value: Not specife: a decrease in score indicates improvement: hicher scores indicate worse outcome
Efficacy outcomes for Systemic Sclerosis
ACR-CRISS score (CRISS score ≥0.6 improvement, \< 0.6 no improvement) and modified CRISS score (rCRISS score) (percentage of patients with at least 3 of the 5 core items of ACR-CRISS improved by a certain percentage (e.g. 25%, except FVC (5%))
Efficacy outcomes for Inflammatory Myopathy
Total Improvement Score (TlS): Min/Max Value: Not specified; an increase in score indicates improvement, higher scores indicate better outcomes.
Peak Plasma Concentration (Cmax) of YTS109
Cmax will be determined using validated quantitative PCR assay in peripheral blood samples.
Time to Peak (Tmax) of YTS109
Time from infusion to observed Cmax (Days) . To evaluate the metabolic characteristics of YTS109
Area under the plasma concentration versus time curve (AUC) of YTS109
Calculated using non-compartmental analysis. To evaluate the metabolic characteristics of YTS109
Efficacy outcomes for SLE
SLE Response index 4 (SR-4) response: Min/Max Value: Not specific: a decrease in score indicates improvement. Higher scores indicate worse outcomes.
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Children's Hospital of Fudan University
Lead sponsor
China Immunotech (Beijing) Biotechnology Co., Ltd.
Collaborator
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