This is an investigator-initiated, single-center, open-label study of C-CAR168, an autologous bi-specific CAR-T therapy targeting CD20 and BCMA, for the treatment of adult patients with central nervous system autoimmune diseases refractory to standard therapy
18 to 70 years old at the time of signing the Informed Consent Form (ICF).
Diagnosed as Multiple Sclerosis (MS)/Neuromyelitis Optica Spectrum Disorders (NMOSD)/Autoimmune Encephalitis(AiE)/Stiff Person Spectrum Disorder(SPSD) according to recognized diagnostic criteria for at least 6 months.
Prior treatment failure with standard therapy.
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.
Sequential
CD20/BCMA-directed CAR-T cells
Biological/VaccineAutologous 2nd generation CD20/BCMA-directed CAR-T cells, single infusion intravenously
Incidence and severity of Adverse Events [Safety and Tolerability]
Incidence and severity of adverse events (AE) and serious adverse events (SAE) within three months following infusion
The subsequent recommended dose of C-CAR168 in patients with central nervous system autoimmune diseases refractory to standard therapy
Based on the assessment of overall safety profile
Incidence and severity of adverse events (AE)
Incidence and severity of adverse events (AE) and serious adverse events (SAE) during the study
MS: No Evidence of Disease Activity-3 (NEDA-3)
Proportion of participants achieving NEDA-3 at 6 months post-infusion and during the study period
MS and NMOSD: Expanded Disability Status Scale (EDSS)
Change from baseline in EDSS at 6 months and 24 months post-infusion. EDSS and its associated functional system (FS) score provide a system for quantifying disability and monitoring changes in the level of disability over time. EDSS consists of 7 FS (visual FS, brainstem FS, pyramidal FS, cerebellar FS, sensory FS, bowel and bladder FS, and cerebral FS) which are used to derive EDSS score ranging from 0 (normal neurological exam) to 10 (death).
MS and NMOSD: MRI
Number of T1 gadolinium-enhancing lesions and new or enlarging T2 lesions, as well as their change from baseline, at 6 months and 24 months post-infusion. Change from baseline in total T2 lesion volume, gray matter volume (GMV), white matter volume (WMV), and brain volume (BV), as well as annualized-brain volume loss (a-BVL), at 6 months and 24 months post-infusion
Serum cytokines changes
Detection of serum cytokines 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)
Changes in CSF CAR DNA copy number and CAR-T cells
Detection of changes in CSF CAR DNA copy number and CAR-T cells by quantitative polymerase chain reaction (qPCR) and flow cytometry
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Huashan Hospital
Lead sponsor
Shanghai AbelZeta Ltd.
Collaborator
This trial is not recruiting at the moment. You can still explore other options:
