This study primarily involves the use of BCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells, for the treatment of patients with refractory autoimmune diseases, aiming to evaluate its safety, tolerability, and dose-limiting toxicities(DLT), and to determine the recommended therapeutic dose for further investigation. Additionally, the study assesses the efficacy of BCT301 cell injection in refractory autoimmune diseases, as well as the pharmacokinetic (PK) and pharmacodynamic (PD) characteristics in study participants.
Voluntarily sign the informed consent form.
Male or female, aged 18-80 years (inclusive), with a body weight ≥40 kg.
Female participants of childbearing potential and male participants with female partners of childbearing potential must use medically approved contraceptive methods or practice abstinence during the treatment period and for at least 6 months after the end of the treatment. Female participants of childbearing potential must have a negative serum human chorionic gonadotropin (HCG) test within 7 days prior to enrollment and must not be breastfeeding.
If the participant is receiving glucocorticoid therapy, the following conditions must be met: the maximum dose at screening and during the screening period is 30 mg/day of prednisone (or equivalent). The dose must have been stable for ≥7 days prior to screening, and adjustments during the screening period must not exceed 5 mg/day of prednisone (or equivalent);
Any medical condition that, in the opinion of the investigator, would contraindicate participation in the study, such as a life-threatening illness.
History of alcohol or substance abuse within the past 24 weeks.
History of malignancy other than B-cell lymphoma.
Presence of infections including human immunodeficiency virus (HIV), hypogammaglobulinemia, T-cell deficiency, syphilis, chronic hepatitis B or C, or severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2).
Single group
BCT301
Biological/VaccineBCT301, an anti-CD19 Chemically induced pluripotent stem cell (CiPSC)-derived CAR-iT cells.Participants will receive a single infusion of BCT301 cell injection at escalating dose levels.Prior to infusion, patients undergo a lymphodepleting conditioning regimen with fludarabine and cyclophosphamide given intravenously for three consecutive days.
Dose-limiting toxicities of BCT301
Adverse events occurring within 28 days post-infusion that were possibly, probably, or definitely related to BCT301 cell injection (per NCI-CTCAE v5.0).
Efficacy of refractory systemic lupus erythematosus（SLE）- The change of SLEDAI-2K
The primary efficacy endpoint will be the change from baseline in the SLEDAI-2K score. The SLEDAI-2K is a validated index for assessing SLE disease activity, with scores ranging from 0 to 105. A higher score indicates worse disease activity.
Efficacy of refractory SLE-The change of PGA
The Physician Global Assessment (PGA) will be used to evaluate overall SLE disease activity. The PGA is a clinician's determination of disease activity based on history and physical exam, independent of laboratory results. It is scored on a scale from 0 to 3, where a higher score indicates worse disease activity.
Efficacy of refractory SLE-The change of SF36
The SF-36 health survey will be employed as a patient-reported outcome to assess health-related quality of life. This 36-item questionnaire generates scores from 0 to 100 across eight health domains and two summary components (Physical and Mental). A lower score indicates a worse health state
Efficacy of refractory SLE-The change of antibodies
Changes in autoantibody titers (e.g., anti-dsDNA) will be assessed as a serological outcome. A higher autoantibody titer reflects more severe underlying immunologic activity
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