This is an open-label, single-arm, dose-escalation study in up to 20 participants with relapsed/refractory Neuromyelitis Optica Spectrum Disorders (NMOSD). The aim is to evaluate the safety and efficacy of the treatment with BAFFR CART.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Anti-BAFFR CART
Incidence of dose-limiting toxicity (DLT)
Incidence of dose-limiting toxicity (DLT) will be evaluated within the first 28 days following BAFFR CART cells infusion.
Time frame: Up to 28 days
Incidence and severity of adverse events
Adverse events will be evaluated following the chemotherapy preparative regimen and infusion of BAFFR CART cells within the first 28 days.
Time frame: Up to 28 days
Proportion of BAFFR CART cells in peripheral blood
The proportion of BAFFR CART cells in peripheral blood will be detected after infusion.
Time frame: Up to 1 years
Annualized relapse rate (ARR)
ARR is defined as the number of attacks divided by the total participant-years after infusion.
Time frame: Up to 1 years
Changes of B cell levels in bone marrow and peripheral blood
The changes of B cell levels in bone marrow and peripheral blood after BAFFR CART infusion.
Time frame: Up to 1 years
Changes of AQP4 antibody titers
Changes of AQP4 antibody titers from baseline over 1 year after BAFFR CART infusion.
Time frame: Up to 1 years
Changes of cytokine in peripheral blood
Changes of cytokine(including IL-6, IFN-γ, TNF-α, and serum BAFF) in peripheral blood from baseline over 1 year after BAFFR CART infusion.
Time frame: Up to 1 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Changes of Expanded Disability Status Scale (EDSS) score
Participant will be considered to have a worsening in overall EDSS score of at least 2 if baseline EDSS score was 0, or at least 1 point if baseline EDSS score is 1 to 5, or at least 0.5 point if baseline EDSS score is 5.5 or more.
Time frame: Up to 1 years
Changes of visual acuity
Corrected visual acuity is determine by Snellen E chart held at a distance of 5 meters.
Time frame: Up to 1 years
Accumulated total active MRI lesions
The changes of T2 lesions and/or enhancing T1 Lesions as detected by brain Magnetic Resonance Imaging (MRI).
Time frame: Up to 1 years