Skip to main content
OpenTrials
Not Yet Recruiting

NCT Number: NCT07592754

The Safety and Efficacy of KSVCBD Injection in 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

Not Yet Recruiting

Trial opening soon.

Get Notified

Key information

Conditions

Neuromyelitis Optica Spectrum Disease (NMOSD) Autoimmune Diseases Autoimmune Diseases of the Nervous System Cranial Nerve Diseases Demyelinating Autoimmune Diseases, CNS Demyelinating Diseases Eye Diseases Immune System Diseases Myelitis, Transverse Nervous System Diseases Neuromyelitis Optica Optic Nerve Diseases Optic Neuritis

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • 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.

Key Exclusion Criteria:

  • 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.

Treatment and study plan

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.

Primary outcomes

  1. Dose limited toxicity (DLT)

    Time frame: Within 28 days post-infusion

    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).

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

    Time frame: Within 24 months post-infusion

    Incidence and severity of AEs and SAEs

  3. Adverse events of special interest (AESI)

    Time frame: Within 24 months post-infusion

    AESI including grade ≥3 Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), and infections

Secondary outcomes

  1. ARR

    Time frame: Within 24 months post-infusion

    Annualized relapse rate (ARR), Time to first relapse, and annualized hospitalization frequency after treatment.

  2. Number of Active lesions

    Time frame: Within 24 months post-infusion

    Change from baseline in the total number of active lesions on MRI after treatment;

  3. EDSS score

    Time frame: Within 24 months post-infusion

    Change from baseline in EDSS score

  4. AQP4 antibody

    Time frame: Within 24 months post-infusion

    Change from baseline in aquaporin-4 (AQP4) antibody level

  5. Visual status

    Time frame: Within 24 months post-infusion

    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.

  6. KSVCBD lentiviral particle concentration

    Time frame: Within 24 months post-infusion

    KSVCBD lentiviral particle concentration in peripheral blood.

  7. CAR-positive T cells

    Time frame: Within 24 months post-infusion

    CAR-positive T cells in peripheral blood.

  8. CAR gene copy number

    Time frame: Within 24 months post-infusion

    CAR gene copy number in peripheral blood

  9. Number of CD19-positive cells

    Time frame: Within 24 months post-infusion

    Number of CD19-positive cells in peripheral blood.

  10. Number of BCMA-positive cells

    Time frame: Within 24 months post-infusion

    Number of BCMA-positive cells in peripheral blood

Study contacts

Contact information is provided by the study sponsor or research team.

Shihui Wei, MD

CONTACT

[email protected]

+86 13910079431

Sponsors and collaborators

Lead sponsor

Chinese PLA General Hospital

Other

Registry information

Official study title

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.

Important dates

Study start
2026
Primary completion
2028
Study completion
2029
First posted
May 18, 2026
Registry last updated
May 18, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.