Neuromyelitis OpticaPhase 1NCT05828212
Evaluate the Safety and Efficacy of CAR-T Cells in the Treatment of R/R Neuromyelitis Optica
A Phase 1 study testing CAR-T cells injection in people with Neuromyelitis Optica, run by Zhejiang University at 1 site in China.
Trial summary
This is a single-arm, open-label, single-center, phase I study. The primary objective is to evaluate the safety of CD19 CAR-T therapy for patients with relapsed or refractory Neuromyelitis Optica, and to evaluate the pharmacokinetics of CD19 CAR-T in patients. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Neuromyelitis Optica
- What is being tested?
- CAR-T cells injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 15 May 2023to 30 Jul 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 1. Age 18-60 and gender unlimited;
- 2. NMOSD diagnosed based on the 2015 NMOSD International Consensus Diagnostic Criteria;
- 3. Diagnostic criteria for AQP4-IgG positive NMOSD Diagnostic criteria for AQP4-IgG positive NMOSD
- At least 1 core clinical feature
- Using reliable methods to detect positive AQP4-IgG (CBA method)
- Exclude other diagnoses. Core clinical features
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- ＯＮ
- Acute myelitis
- Posterior region syndrome, unexplained paroxysmal hiccup, nausea, and vomiting
- Other brainstem syndromes
- Symptomatic episodic sleeping sickness, diencephalic syndrome, brain MRI with NMOSD characteristic diencephalic lesions
- Cerebral syndrome with NMOSD characteristic brain lesions
- 4. Corticosteroids combined with immunosuppressants (azathioprine or mycophenolate mofetil or rituximab) still relapse after treatment；
- 5. At least 2 relapses within the past 12 months or at least 3 relapses within the past 24 months, and at least 1 recurrence within the 12 months prior to screening；
- 6. The estimated survival time is more than 12 weeks;
- 7. Women of childbearing age who have negative urine pregnancy test before medication administration and agree to take effective contraceptive measures during the trial period until the last follow-up
Who cannot join
- 1. Epilepsy history or other central nervous system disease;
- 2. Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythm ia in the past;
- 3. Pregnant (or lactating) women;
- 4. Patients with severe active infections;
- 5. Active infection of hepatitis B virus or hepatitis C virus;
- 6. Systemic steroids have used in the 4 weeks before participating in the treatment (except recently or currently using inhaled steroids);
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- 7. Those who have used any gene therapy products before;
- 8. The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
- 9. Serum creatinine > 2.5mg/dl or ALT / AST > 3 times ULN or bilirubin > 2.0mg/dl;
- 10. Those who suffer from other uncontrolled diseases are not suitable to join the study;
- 11. HIV infection;
- 12. Any situation that the researchers believe may increase the risk of patients or interfere with the test results.
Trial locations and contacts
1 site in 1 country.
China1 site
The first affiliated hospital of medical college of zhejiang university
Hangzhou, Zhejiang, 310000
About this study
Official title: A Study on the Safety and Efficacy of Chimeric Antigen Receptor T Cells in the Treatment of Recurrent/Refractory Neuromyelitis Optica
This is a single-arm, open-label, single-center, phase I study. The primary objective is to evaluate the safety of CD19 CAR-T therapy for patients with relapsed or refractory Neuromyelitis Optica, and to evaluate the pharmacokinetics of CD19 CAR-T in patients.
- Conditions
- Neuromyelitis Optica
- Being tested
- CAR-T cells injection
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CAR-T cells injectionDrug
CAR-T cells in the treatment of R/R neuromyelitis optica
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group | Experimental | Recurrent/Refractory Neuromyelitis Optica Receives: Drug: CAR-T cells injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose limited toxicity (DLT)
Dose limited toxicity
Measured: From date of initial treatment to Day 28 post CAR-T infusion.
AE and SAE
Adverse event and serious adverse event
Measured: From admission to the end of the follow-up, up to 2 years
Maximum tolerable dose
Maximum tolerable dose
Measured: From date of initial treatment to Day 28 post CAR-T infusion.
Secondary outcomes (7)
Changes in serum AQP4-IgG titer after infusion
Changes in serum AQP4-IgG titer after infusion
Measured: days 7, 14, 21, 28 and 90
Annual recurrence rate (ARR) of NMOSD
Annual recurrence rate (ARR) of NMOSD
Measured: From admission to the end of the follow-up, up to 2 years
Changes in the expanded disability status scale (EDSS) score from baseline
EDSS:0 (normal neurological exam) to 10 (death due to MS),Higher scores indicate greater disability (worse outcome)，Score reduction suggests functional improvement
Measured: days 7, 14, 21, 28 ,56 and 90
MRI active lesions
Proportion of subjects with ≥1 active lesions at Day 90
Measured: days 90
Changes in optimal corrected vision
Changes in optimal corrected vision (Log MAR)
Measured: days 28 and 90
Changes of nerve fiber layer around the retinal papilla(pRNFL)
Change in RNFL by optical coherence tomography over trial
Measured: 2 years
Changes in the plexiform layer of macular ganglion cells (mGCIPL)
Proportion of subjects with significant mGCIPL thickness changes
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2023
- Study start15 May 2023
- Primary completion30 Jul 2025
- Study completion30 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis OpticaStudies of the same condition, open and under way.
- More trials run by Zhejiang UniversityThe 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zhejiang University
- Sponsor type
- University, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Responsible party
- Principal investigator, He Huang, Zhejiang University
- Investigators
- He Huang, MD, Principal investigator, First Affiliated Hospital of Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05828212
- Sponsor's protocol ID
- TXB2023005
- Status last verified
- Jul 2025
- First submitted
- 12 Apr 2023
- Last updated
- 11 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study on the Safety and Efficacy of Chimeric Antigen Receptor T Cells in the Treatment of Recurrent/Refractory Neuromyelitis Optica
- ClinicalTrials.gov ID
- NCT05828212
- Acronym / sponsor ID
- TXB2023005
- Conditions
- Neuromyelitis Optica
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Zhejiang UniversityUniversity, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Study start
- 15 May 2023 (actual)
- Primary completion
- 30 Jul 2025 (actual)
- Study completion
- 30 Jul 2025 (actual)
- First posted
- 25 Apr 2023 (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.
This is a single-arm, open-label, single-center, phase I study. The primary objective is to evaluate the safety of CD19 CAR-T therapy for patients with relapsed or refractory Neuromyelitis Optica, and to evaluate the pharmacokinetics of CD19 CAR-T in patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Group | Experimental | Drug: CAR-T cells injectionRecurrent/Refractory Neuromyelitis Optica |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (17 listed)
- 1. Age 18-60 and gender unlimited;
- 2. NMOSD diagnosed based on the 2015 NMOSD International Consensus Diagnostic Criteria;
- 3. Diagnostic criteria for AQP4-IgG positive NMOSD Diagnostic criteria for AQP4-IgG positive NMOSD
- <!-- -->
- 4. Corticosteroids combined with immunosuppressants (azathioprine or mycophenolate mofetil or rituximab) still relapse after treatment；
Key exclusion criteria (12 listed)
- 1. Epilepsy history or other central nervous system disease;
- 2. Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythm ia in the past;
- 3. Pregnant (or lactating) women;
- 4. Patients with severe active infections;
- 5. Active infection of hepatitis B virus or hepatitis C virus;
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 |
|---|---|---|
| CAR-T cells injectionCAR-T cells in the treatment of R/R neuromyelitis optica | Drug | Treatment Group |
Primary outcome
Dose limited toxicity (DLT)
Time frame: From date of initial treatment to Day 28 post CAR-T infusion.
Dose limited toxicity
AE and SAE
Time frame: From admission to the end of the follow-up, up to 2 years
Adverse event and serious adverse event
Maximum tolerable dose
Time frame: From date of initial treatment to Day 28 post CAR-T infusion.
Maximum tolerable dose
7 secondary outcomes
Changes in serum AQP4-IgG titer after infusion
Time frame: days 7, 14, 21, 28 and 90
Annual recurrence rate (ARR) of NMOSD
Time frame: From admission to the end of the follow-up, up to 2 years
Changes in the expanded disability status scale (EDSS) score from baseline
Time frame: days 7, 14, 21, 28 ,56 and 90
MRI active lesions
Time frame: days 90
Changes in optimal corrected vision
Time frame: days 28 and 90
Changes of nerve fiber layer around the retinal papilla(pRNFL)
Time frame: 2 years
Changes in the plexiform layer of macular ganglion cells (mGCIPL)
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2025.
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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. This study is not recruiting at the moment.
He Huang, MD principal investigator
First Affiliated Hospital of Zhejiang University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CAR-T cells injection in people with Neuromyelitis Optica, run by Zhejiang University 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 17 inclusion and 12 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: finished, as of the sponsor's last update on 11 Aug 2025. 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?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose limited toxicity (DLT) (time frame: From date of initial treatment to Day 28 post CAR-T 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 11 Aug 2025.