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

NCT07592754 | Not Yet Recruiting Early Phase 1

• 1 other identifier: KSVCBD-R103
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Neuromyelitis Optica Spectrum Disease (NMOSD)

Keywords

Neuromyelitis Optica Spectrum Disorders

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (10)

• ARR

  Within 24 months post-infusion

• Number of Active lesions

  Within 24 months post-infusion

• EDSS score

  Within 24 months post-infusion

• AQP4 antibody

  Within 24 months post-infusion

• Visual status

  Within 24 months post-infusion

Study Arms (1)

AQP4-positive Neuromyelitis Optica Spectrum Disorders

EXPERIMENTAL

Biological: KSVCBD injection

Interventions

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.

AQP4-positive Neuromyelitis Optica Spectrum Disorders

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must meet the 2015 International Consensus Diagnostic Criteria for Neuromyelitis Optica Spectrum Disorder (NMOSD) and test positive for AQP4 antibodies.
• Documented evidence of at least 1 relapse within 12 months before signing the informed consent form.
• The Expanded Disability Status Scale (EDSS) score ≤ 8.
• Female participants of childbearing potential must present a negative pregnancy test at screening and agree to use effective contraception throughout the study period.
• Informed consent must be obtained from the patient or their legal representative, with a signed consent form must be provided.

You may not qualify if:

• Viral infections: Known HIV, active HBV, or active HCV.
• Pregnant or breastfeeding women.
• History of other autoimmune diseases requiring immunosuppressive therapy.
• Use of any live vaccines against infectious disease within 6 weeks before enrollment.
• History of bone marrow/hematopoietic stem cell or solid organ transplantation.
• Patients deemed unsuitable for participation by the investigator.

Sponsors & Collaborators

• Chinese PLA General Hospital: lead

Study Sites (1)

Chinese PLA General Hospital

Beijing, Beijing Municipality, 100853, China

Location

MeSH Terms

Conditions

Neuromyelitis Optica

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Shihui Wei, MD

CONTACT

+86 13910079431; weishihui706@hotmail.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief Physician and Professor

Study Record Dates

• First Submitted: May 12, 2026
• First Posted: May 18, 2026
• Study Start: May 15, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): March 31, 2029
• Last Updated: May 18, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)