This is an investigator-initiated, multicenter, open-label study of C-CAR168, an autologous bi-specific CAR-T therapy targeting CD20 and BCMA, for the treatment of adult patients with autoimmune diseases refractory to standard therapy
18 to 70 years old at the time of signing the Informed Consent Form (ICF).
Diagnosed as SLE/Immune-Mediated Necrotizing Myopathy (IMNM)/Neuromyelitis Optica Spectrum Disorders (NMOSD)/Multiple Sclerosis (MS)/Myasthenia Gravis (MG)/Systemic Sclerosis (SSc) according to recognized diagnostic criteria for at least 6 months.
Remains disease active or relapses after treatment with standard of care therapy for at least 8 weeks with the dose stable for more than 2 weeks; patients should have been treated with at least two immunosuppressants (including immunosuppressants, biologics, and disease-modifying drug (DMD) ).
Adequate bone marrow, coagulation, cardiopulmonary, liver and renal function.
Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), Human Immunodeficiency Virus (HIV), Treponema Pallidum (TP) positive, Cytomegalovirus (CMV) DNA positive, Epstein-Barr Virus (EBV) DNA positive.
Uncontrolled active infection.
Live vaccine injection within 4 weeks prior to signing the ICF.
Major organ transplantation history or bone marrow/hematopoietic stem cell transplantation history.
Single group
CD20/BCMA-directed CAR-T cells
Biological/VaccineAutologous 2nd generation CD20/BCMA-directed CAR-T cells, single infusion intravenously
Incidence of Adverse Events [Safety and Tolerability]
Incidence of any adverse events (AEs), including dose limiting toxicities (DLTs)
The subsequent recommended dose of C-CAR168 in patients with autoimmune diseases refractory to standard therapy
Based on the assessment of dose-limiting toxicities (DLTs) rates and overall safety profile
The proportion of subjects who achieved remission at 6 months (6M)
The proportion of subjects who achieved remission during the main study period
The proportion of subjects who experienced relapse during the main study period
Time to response (TTR)
The time from the date of C-CAR168 infusion to the first documented remission
Serum cytokines (including Interleukin (IL)-2, IL-4, IL-6, IL-10, Tumor Necrosis Factor (TNF)-α, Interferon (IFN)-γ) changes
Detection of serum cytokines (including IL-2, IL-4, IL-6, IL-10, TNF-α, IFN-γ) changes over time by flow cytometry
Soluble BCMA changes in peripheral blood
Detection of soluble BCMA changes in peripheral blood by Enzyme Linked ImmunoSorbent Assay (ELISA)
RNA changes in peripheral blood
Detection of RNA changes in peripheral blood by RNA sequencing
Protective antibodies changes in peripheral blood
Detection of protective antibodies changes in peripheral blood by ELISA
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RenJi Hospital
Lead sponsor
AbelZeta Inc.
Collaborator
