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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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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

Other trials in China

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.

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 groups in NCT05828212
Study groupRoleWhat they receive
Treatment GroupExperimentalRecurrent/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.

  1. Dose limited toxicity (DLT)

    Dose limited toxicity

    Measured: From date of initial treatment to Day 28 post CAR-T infusion.

  2. AE and SAE

    Adverse event and serious adverse event

    Measured: From admission to the end of the follow-up, up to 2 years

  3. Maximum tolerable dose

    Maximum tolerable dose

    Measured: From date of initial treatment to Day 28 post CAR-T infusion.

Secondary outcomes (7)

  1. Changes in serum AQP4-IgG titer after infusion

    Changes in serum AQP4-IgG titer after infusion

    Measured: days 7, 14, 21, 28 and 90

  2. 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

  3. 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

  4. MRI active lesions

    Proportion of subjects with ≥1 active lesions at Day 90

    Measured: days 90

  5. Changes in optimal corrected vision

    Changes in optimal corrected vision (Log MAR)

    Measured: days 28 and 90

  6. Changes of nerve fiber layer around the retinal papilla(pRNFL)

    Change in RNFL by optical coherence tomography over trial

    Measured: 2 years

  7. 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.

  1. First posted25 Apr 2023
  2. Study start15 May 2023
  3. Primary completion30 Jul 2025
  4. Study completion30 Jul 2025

Resources

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)
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

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?

On the ClinicalTrials.gov record for NCT05828212.

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.

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