The Safety and Efficacy of KSVCBD Injection in Neuromyelitis Optica Spectrum Disorder.

Early Phase 1Not Yet RecruitingNCT07592754

Chinese PLA General Hospital18 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Neuromyelitis Optica Spectrum Disease (NMOSD)

Interventions

KSVCBD injectionBIOLOGICAL

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.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: 1. Patients must meet the 2015 International Consensus Diagnostic Criteria for Neuromyelitis Optica Spectrum Disorder (NMOSD) and test positive for AQP4 antibodies. 2. Documented evidence of at least 1 relapse within 12 months before signing the informed consent form. 3. The Expanded Disability Status Scale (EDSS) score ≤ 8. 4. Female participants of childbearing potential must present a negative pregnancy test at screening and agree to use effective contraception throughout the study period. 5. Informed consent must be obtained from the patient or their legal representative, with a signed consent form must be provided. Key Exclusion Criteria: 1. Viral infections: Known HIV, active HBV, or active HCV. 2. Pregnant or breastfeeding women. 3. History of other autoimmune diseases requiring immunosuppressive therapy. 4. Use of any live vaccines against infectious disease within 6 weeks before enrollment. 5. History of bone marrow/hematopoietic stem cell or solid organ transplantation. 6. Patients deemed unsuitable for participation by the investigator.

Locations (1)

Chinese PLA General Hospital

Beijing, Beijing Municipality, China

Outcomes

Primary Outcomes

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).

Time frame: Within 28 days post-infusion

Adverse events (AEs) and serious adverse events (SAEs)

Incidence and severity of AEs and SAEs