eligibility_summary
Include: ≥5 yrs, JDM with refractory course (failed steroids+≥2 IS or rapid organ involvement, calcinosis/rash, or persistent weakness with MRI/CMAS + core abnormalities), SRP/HMGCR+ IMNM, adequate heart/kidney/liver/lung function. Exclude: prior CAR‑T, immunodeficiency, active infection or cancer, serious cardiac disease, other chronic steroid/IS need, recent transplant/GVHD, viral hepatitis/HIV/syphilis, live vaccine <4 wks, pregnancy, other trial <3 mo, unsuitable.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
ai
ai_summary
Intervention: Anti-CD19 Universal CAR-T (UCAR-T) cells—an allogeneic, gene-edited cellular immunotherapy. Patients receive a single IV infusion after lymphodepletion. Mechanism of action: T lymphocytes engineered with a chimeric antigen receptor specific for CD19 recognize and kill B cells via T-cell cytotoxic pathways, producing deep depletion of CD19+ B cells/plasmablasts to reduce autoantibody production and rebalance immune function in refractory juvenile dermatomyositis. Targets: CD19-expressing B-lineage cells (naive/memory B cells, plasmablasts), downstream humoral/autoantibody pathways, germinal center activity, and immune reconstitution. Design: open-label, single-arm, 3+3 dose escalation (1×10^7–6×10^7 cells/kg) assessing safety, PK/PD, and efficacy.