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Completed

NCT Number: NCT05828212

Evaluate the Safety and Efficacy of CAR-T Cells in the Treatment of R/R 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.

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Key information

Age range

18 year–60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

The first affiliated hospital of medical college of zhejiang university

Hangzhou, Zhejiang, 310000, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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);
  • 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.

Treatment and study plan

CAR-T cells injection

Drug

CAR-T cells in the treatment of R/R neuromyelitis optica

Primary outcomes

  1. Dose limited toxicity (DLT)

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

    Dose limited toxicity

  2. AE and SAE

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

    Adverse event and serious adverse event

  3. Maximum tolerable dose

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

    Maximum tolerable dose

Secondary outcomes

  1. Changes in serum AQP4-IgG titer after infusion

    Time frame: days 7, 14, 21, 28 and 90

    Changes in serum AQP4-IgG titer after infusion

  2. Annual recurrence rate (ARR) of NMOSD

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

    Annual recurrence rate (ARR) of NMOSD

  3. Changes in the expanded disability status scale (EDSS) score from baseline

    Time frame: days 7, 14, 21, 28 ,56 and 90

    EDSS:0 (normal neurological exam) to 10 (death due to MS),Higher scores indicate greater disability (worse outcome),Score reduction suggests functional improvement

  4. MRI active lesions

    Time frame: days 90

    Proportion of subjects with ≥1 active lesions at Day 90

  5. Changes in optimal corrected vision

    Time frame: days 28 and 90

    Changes in optimal corrected vision (Log MAR)

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

    Time frame: 2 years

    Change in RNFL by optical coherence tomography over trial

  7. Changes in the plexiform layer of macular ganglion cells (mGCIPL)

    Time frame: 2 years

    Proportion of subjects with significant mGCIPL thickness changes

Sponsors and collaborators

Lead sponsor

Zhejiang University

Other

Collaborators

  • Yake Biotechnology Ltd.

Registry information

Official study title

A Study on the Safety and Efficacy of Chimeric Antigen Receptor T Cells in the Treatment of Recurrent/Refractory Neuromyelitis Optica

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Apr 25, 2023
Registry last updated
Aug 11, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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