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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Participant Group | Experimental | BAFFR CART cells |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Anti-BAFFR CART | Drug | Anti-BAFFR CART |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | Up to 28 days |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of BAFFR CART cells in peripheral blood | The proportion of BAFFR CART cells in peripheral blood will be detected after infusion. | Up to 1 years |
| Annualized relapse rate (ARR) | ARR is defined as the number of attacks divided by the total participant-years after infusion. |
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Inclusion Criteria:
Exclusion Criteria:
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| ID | Term |
|---|---|
| D009471 | Neuromyelitis Optica |
| ID | Term |
|---|---|
| D009188 | Myelitis, Transverse |
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
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| 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. | Up to 1 years |
| Changes of AQP4 antibody titers | Changes of AQP4 antibody titers from baseline over 1 year after BAFFR CART infusion. | 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. | Up to 1 years |
| 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. | Up to 1 years |
| Changes of visual acuity | Corrected visual acuity is determine by Snellen E chart held at a distance of 5 meters. | 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). | Up to 1 years |
| D009902 | Optic Neuritis |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D003711 | Demyelinating Diseases |
| D005128 | Eye Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |