The Safety and Efficacy of KSVCBD Injection in Neuromyelitis Optica Spectrum Disorder.
12. maj 2026 opdateret af: Shihui Wei, Chinese PLA General Hospital
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
Studieoversigt
Status
Ikke rekrutterer endnu
Betingelser
Intervention / Behandling
Detaljeret beskrivelse
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.
Undersøgelsestype
Interventionel
Tilmelding (Anslået)
18
Fase
- Tidlig fase 1
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiekontakt
- Navn: shihui Wei, MD
- Telefonnummer: +86 13910079431
- E-mail: weishihui706@hotmail.com
Studiesteder
-
-
Beijing Municipality
-
Beijing, Beijing Municipality, Kina, 100853
- Chinese PLA General Hospital
-
Kontakt:
- shihui Wei, MD
- Telefonnummer: +86 13910079431
- E-mail: weishihui706@hotmail.com
-
-
Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Ingen
Beskrivelse
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.
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: AQP4-positive Neuromyelitis Optica Spectrum Disorders
|
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.
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Dose limited toxicity (DLT)
Tidsramme: 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).
|
Within 28 days post-infusion
|
|
Adverse events (AEs) and serious adverse events (SAEs)
Tidsramme: Within 24 months post-infusion
|
Incidence and severity of AEs and SAEs
|
Within 24 months post-infusion
|
|
Adverse events of special interest (AESI)
Tidsramme: Within 24 months post-infusion
|
AESI including grade ≥3 Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), and infections
|
Within 24 months post-infusion
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
ARR
Tidsramme: Within 24 months post-infusion
|
Annualized relapse rate (ARR), Time to first relapse, and annualized hospitalization frequency after treatment.
|
Within 24 months post-infusion
|
|
Number of Active lesions
Tidsramme: Within 24 months post-infusion
|
Change from baseline in the total number of active lesions on MRI after treatment;
|
Within 24 months post-infusion
|
|
EDSS score
Tidsramme: Within 24 months post-infusion
|
Change from baseline in EDSS score
|
Within 24 months post-infusion
|
|
AQP4 antibody
Tidsramme: Within 24 months post-infusion
|
Change from baseline in aquaporin-4 (AQP4) antibody level
|
Within 24 months post-infusion
|
|
Visual status
Tidsramme: 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.
|
Within 24 months post-infusion
|
|
KSVCBD lentiviral particle concentration
Tidsramme: Within 24 months post-infusion
|
KSVCBD lentiviral particle concentration in peripheral blood.
|
Within 24 months post-infusion
|
|
CAR-positive T cells
Tidsramme: Within 24 months post-infusion
|
CAR-positive T cells in peripheral blood.
|
Within 24 months post-infusion
|
|
CAR gene copy number
Tidsramme: Within 24 months post-infusion
|
CAR gene copy number in peripheral blood
|
Within 24 months post-infusion
|
|
Number of CD19-positive cells
Tidsramme: Within 24 months post-infusion
|
Number of CD19-positive cells in peripheral blood.
|
Within 24 months post-infusion
|
|
Number of BCMA-positive cells
Tidsramme: Within 24 months post-infusion
|
Number of BCMA-positive cells in peripheral blood
|
Within 24 months post-infusion
|
Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Anslået)
15. maj 2026
Primær færdiggørelse (Anslået)
31. december 2028
Studieafslutning (Anslået)
31. marts 2029
Datoer for studieregistrering
Først indsendt
12. maj 2026
Først indsendt, der opfyldte QC-kriterier
12. maj 2026
Først opslået (Faktiske)
18. maj 2026
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
18. maj 2026
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
12. maj 2026
Sidst verificeret
1. maj 2026
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- KSVCBD-R103
Plan for individuelle deltagerdata (IPD)
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