Using CAR-T cells to treat relapsed or refractory Neuromyelitis Optica

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

This study is testing if a new treatment using CAR-T cells can help people with relapsed or hard-to-treat Neuromyelitis Optica feel better.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Ages: 18 Years to 60 Years
• Sex: All
• Sponsor: Zhejiang University (Academic / other)
• Drugs / interventions: rituximab, CAR-T
• Locations: 1 site (Hangzhou, Zhejiang)
• Trial ID: NCT05828212 on ClinicalTrials.gov

What this trial studies

This is a single-arm, open-label, phase I clinical trial that evaluates the safety and efficacy of CD19 CAR-T cell therapy for patients diagnosed with relapsed or refractory Neuromyelitis Optica. The study aims to assess the pharmacokinetics of the CAR-T cells while monitoring for any adverse effects. Participants will receive injections of CD19 CAR-T cells and will be closely monitored throughout the trial. The study is conducted at a single center, the First Affiliated Hospital of Zhejiang University.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18-60 with a confirmed diagnosis of AQP4-IgG positive Neuromyelitis Optica who have experienced multiple relapses despite standard treatments.

Not a fit: Patients who do not have AQP4-IgG positive Neuromyelitis Optica or those who have not experienced relapses despite treatment may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a novel therapeutic option for patients suffering from relapsed or refractory Neuromyelitis Optica.

How similar studies have performed: While CAR-T cell therapy has shown promise in other conditions, this specific application for Neuromyelitis Optica is novel and has not been extensively tested in prior studies.

Eligibility criteria

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

  1. At least 1 core clinical feature
  2. Using reliable methods to detect positive AQP4-IgG (CBA method)
  3. Exclude other diagnoses. Core clinical features

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  1. ＯＮ
  2. Acute myelitis
  3. Posterior region syndrome, unexplained paroxysmal hiccup, nausea, and vomiting
  4. Other brainstem syndromes
  5. Symptomatic episodic sleeping sickness, diencephalic syndrome, brain MRI with NMOSD characteristic diencephalic lesions
  6. 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.

Where this trial is running

Hangzhou, Zhejiang

• The first affiliated hospital of medical college of zhejiang university — Hangzhou, Zhejiang, China (Recruiting)

Study contacts

• Principal investigator: He Huang, MD — Zhejiang University
• Study coordinator: He Huang, MD

Conditions Neuromyelitis Optica