The Safety and Efficacy of KSVCBD Injection in Neuromyelitis Optica Spectrum Disorder.
Trial statusNot yet recruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-65
SponsorChinese PLA General Hospital
About this trial
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
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
KSVCBD injection
Biological/VaccineKSVCBD 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.
Treatment groups
18 Participants
are divided into 1 treatment groupGroup A: AQP4-positive Neuromyelitis Optica Spectrum DisordersExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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-infusion2
Adverse events (AEs) and serious adverse events (SAEs)
Incidence and severity of AEs and SAEs
Time frame
Within 24 months post-infusion3
Adverse events of special interest (AESI)
AESI including grade ≥3 Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), and infections
Time frame
Within 24 months post-infusionSecondary outcomes
1
ARR
Annualized relapse rate (ARR), Time to first relapse, and annualized hospitalization frequency after treatment.
Time frame
Within 24 months post-infusion2
Number of Active lesions
Change from baseline in the total number of active lesions on MRI after treatment;
Time frame
Within 24 months post-infusion3
EDSS score
Change from baseline in EDSS score
Time frame
Within 24 months post-infusion4
AQP4 antibody
Change from baseline in aquaporin-4 (AQP4) antibody level
Time frame
Within 24 months post-infusionSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov
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