Neuromyelitis Optica Spectrum Disease (NMOSD)Early Phase 1NCT07592754
The Safety and Efficacy of KSVCBD Injection in Neuromyelitis Optica Spectrum Disorder.
A Early Phase 1 study testing KSVCBD injection in people with Neuromyelitis Optica Spectrum Disease (NMOSD), run by Chinese PLA General Hospital at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disease (NMOSD)
- What is being tested?
- KSVCBD injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 15 May 2026to 31 Mar 2029
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Chinese PLA General Hospital
Beijing, Beijing Municipality, 100853
shihui Wei, MD, +86 13910079431, weishihui706@hotmail.com
About this study
Official 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.
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
Read the 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.
- Being tested
- KSVCBD injection
- Keywords
- Neuromyelitis Optica Spectrum Disorders
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KSVCBD injectionBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| AQP4-positive Neuromyelitis Optica Spectrum Disorders | Experimental | Not described Receives: Biological: KSVCBD injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Within 28 days post-infusion
Adverse events (AEs) and serious adverse events (SAEs)
Incidence and severity of AEs and SAEs
Measured: 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
Measured: Within 24 months post-infusion
Secondary outcomes (10)
ARR
Annualized relapse rate (ARR), Time to first relapse, and annualized hospitalization frequency after treatment.
Measured: Within 24 months post-infusion
Number of Active lesions
Change from baseline in the total number of active lesions on MRI after treatment;
Measured: Within 24 months post-infusion
EDSS score
Change from baseline in EDSS score
Measured: Within 24 months post-infusion
AQP4 antibody
Change from baseline in aquaporin-4 (AQP4) antibody level
Measured: 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.
Measured: Within 24 months post-infusion
KSVCBD lentiviral particle concentration
KSVCBD lentiviral particle concentration in peripheral blood.
Measured: Within 24 months post-infusion
CAR-positive T cells
CAR-positive T cells in peripheral blood.
Measured: Within 24 months post-infusion
CAR gene copy number
CAR gene copy number in peripheral blood
Measured: Within 24 months post-infusion
Number of CD19-positive cells
Number of CD19-positive cells in peripheral blood.
Measured: Within 24 months post-infusion
Number of BCMA-positive cells
Number of BCMA-positive cells in peripheral blood
Measured: Within 24 months post-infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2026
- Study start15 May 2026 (estimated)
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis Optica Spectrum Disease (NMOSD)Studies of the same condition, open and under way.
- More trials run by Chinese PLA General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chinese PLA General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Shihui Wei, Chinese PLA General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07592754
- Sponsor's protocol ID
- KSVCBD-R103
- Status last verified
- May 2026
- First submitted
- 12 May 2026
- Last updated
- 18 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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.
- ClinicalTrials.gov ID
- NCT07592754
- Acronym / sponsor ID
- KSVCBD-R103
- Keywords
- Neuromyelitis Optica Spectrum Disorders
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Chinese PLA General HospitalUniversity, hospital or other
- Study start
- 15 May 2026 (estimated)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Mar 2029 (estimated)
- First posted
- 18 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AQP4-positive Neuromyelitis Optica Spectrum Disorders | Experimental | Biological: KSVCBD injection |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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 (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KSVCBD injectionKSVCBD 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. | Biological | AQP4-positive Neuromyelitis Optica Spectrum Disorders |
Primary outcome
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).
Adverse events (AEs) and serious adverse events (SAEs)
Time frame: Within 24 months post-infusion
Incidence and severity of AEs and SAEs
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
10 secondary outcomes
ARR
Time frame: Within 24 months post-infusion
Number of Active lesions
Time frame: Within 24 months post-infusion
EDSS score
Time frame: Within 24 months post-infusion
AQP4 antibody
Time frame: Within 24 months post-infusion
Visual status
Time frame: Within 24 months post-infusion
KSVCBD lentiviral particle concentration
Time frame: Within 24 months post-infusion
CAR-positive T cells
Time frame: Within 24 months post-infusion
CAR gene copy number
Time frame: Within 24 months post-infusion
Number of CD19-positive cells
Time frame: Within 24 months post-infusion
Number of BCMA-positive cells
Time frame: Within 24 months post-infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing KSVCBD injection in people with Neuromyelitis Optica Spectrum Disease (NMOSD), run by Chinese PLA General Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 18 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Shihui Wei, MD (+86 13910079431, weishihui706@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose limited toxicity (DLT) (time frame: Within 28 days post-infusion). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2026.