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

An Early Exploratory Clinical Study on the Safety and Efficacy of KSVCBD Injection in the Treatment of Relapsed/Refractory AQP4 Antibody-Positive Neuromyelitis Optica Spectrum Disorder.

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

Study Overview

• Status: Not yet recruiting
• Conditions: Neuromyelitis Optica Spectrum Disease (NMOSD)
• Intervention / Treatment: Biological: KSVCBD injection

Detailed Description

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
• Enrollment (Estimated): 18
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: shihui Wei, MD; Phone Number: +86 13910079431; Email: weishihui706@hotmail.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100853
      • Chinese PLA General Hospital
      • Contact: shihui Wei, MD; Phone Number: +86 13910079431; Email: weishihui706@hotmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AQP4-positive Neuromyelitis Optica Spectrum Disorders	Biological: KSVCBD injection; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Secondary Outcome Measures

Outcome Measure	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;	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

Collaborators and Investigators

• Sponsor: Chinese PLA General Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): May 15, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): March 31, 2029

Study Registration Dates

• First Submitted: May 12, 2026
• First Submitted That Met QC Criteria: May 12, 2026
• First Posted (Actual): May 18, 2026

Study Record Updates

• Last Update Posted (Actual): May 18, 2026
• Last Update Submitted That Met QC Criteria: May 12, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Neuromyelitis Optica Spectrum Disorders
• Additional Relevant MeSH Terms: Nervous System Diseases; Autoimmune Diseases; Immune System Diseases; Eye Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Myelitis, Transverse; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Neuromyelitis Optica
• Other Study ID Numbers: KSVCBD-R103

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No