Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
KSVCBD injection is an in vivo Chimeric Antigen Receptor T-Cell (CAR-T cell) therapy product. This is a multi-center, single-arm, open-label, early exploratory clinical study. The objective of this study is to evaluate the safety and preliminary efficacy of KSVCBD injection in AQP4-positive Neuromyelitis Optica Spectrum Disorder
KSVCBD-R103 is an open-label study in subjects with NMOSD. The dose-escalation will be conducted to evaluate the safety and preliminary efficacy of KSVCBD. This study consists of a screening period, a treatment period and a follow-up period. During treatment period, the subjects will receive single-dose of KSVCBD injection. The duration of participation for each subject will be approximately 104 weeks.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AQP4-positive Neuromyelitis Optica Spectrum Disorders | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| KSVCBD injection | Biological | KSVCBD injection is a CD19/BCMA-targeted in vivo-edited CAR-T cell injection. Three dose levels are predefined, and KSVCBD will be dose-escalated per the protocol-specified doses. |
| Measure | Description | Time Frame |
|---|---|---|
| Dose limited toxicity (DLT) | DLT is defined as any of the predefined adverse events (AEs) related to KSVCBD occurring within 28 days after KSVCBD infusion (CRS and ICANS will be graded according to the ASTCT 2019 criteria, and other AEs will be evaluated using CTCAE v6.0). | Within 28 days post-infusion |
| Adverse events (AEs) and serious adverse events (SAEs) | Incidence and severity of AEs and SAEs | Within 24 months post-infusion |
| Adverse events of special interest (AESI) | AESI including grade ≥3 Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), and infections | Within 24 months post-infusion |
| Measure | Description | Time Frame |
|---|---|---|
| ARR | Annualized relapse rate (ARR), Time to first relapse, and annualized hospitalization frequency after treatment. | Within 24 months post-infusion |
| Number of Active lesions | Change from baseline in the total number of active lesions on MRI after treatment; |
Not provided
Key Inclusion Criteria:
Key Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Shihui Wei, MD | Contact | +86 13910079431 | weishihui706@hotmail.com |
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Chinese PLA General Hospital | Beijing | Beijing Municipality | 100853 | China |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| 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 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Within 24 months post-infusion |
| EDSS score | Change from baseline in EDSS score | Within 24 months post-infusion |
| AQP4 antibody | Change from baseline in aquaporin-4 (AQP4) antibody level | Within 24 months post-infusion |
| Visual status | Change from baseline in visual acuity, visual field, and optical coherence tomography [OCT, including retinal nerve fiber layer (RNFL) and ganglion cell layer (GCL)] after treatment. | Within 24 months post-infusion |
| KSVCBD lentiviral particle concentration | KSVCBD lentiviral particle concentration in peripheral blood. | Within 24 months post-infusion |
| CAR-positive T cells | CAR-positive T cells in peripheral blood. | Within 24 months post-infusion |
| CAR gene copy number | CAR gene copy number in peripheral blood | Within 24 months post-infusion |
| Number of CD19-positive cells | Number of CD19-positive cells in peripheral blood. | Within 24 months post-infusion |
| Number of BCMA-positive cells | Number of BCMA-positive cells in peripheral blood | Within 24 months post-infusion |
| D009902 | Optic Neuritis |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D003711 | Demyelinating Diseases |
| D005128 | Eye Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |