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

NCT05828212 | Completed Phase 1

• 1 other identifier: TXB2023005
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Neuromyelitis Optica

Outcome Measures

Primary Outcomes (3)

• Dose limited toxicity (DLT)

  Dose limited toxicity

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

• AE and SAE

  Adverse event and serious adverse event

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

• Maximum tolerable dose

  Maximum tolerable dose

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

Secondary Outcomes (7)

• Changes in serum AQP4-IgG titer after infusion

  days 7, 14, 21, 28 and 90

• Annual recurrence rate (ARR) of NMOSD

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

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

  days 7, 14, 21, 28 ,56 and 90

• MRI active lesions

  days 90

• Changes in optimal corrected vision

  days 28 and 90

Study Arms (1)

Treatment Group

EXPERIMENTAL

Recurrent/Refractory Neuromyelitis Optica

Drug: CAR-T cells injection

Interventions

CAR-T cells injection DRUG

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

Treatment Group

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• \. Age 18-60 and gender unlimited;
• \. NMOSD diagnosed based on the 2015 NMOSD International Consensus Diagnostic Criteria;
• \. 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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• 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
• \. Corticosteroids combined with immunosuppressants (azathioprine or mycophenolate mofetil or rituximab) still relapse after treatment；
• \. 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；
• \. The estimated survival time is more than 12 weeks;

You may not qualify if:

• \. Epilepsy history or other central nervous system disease;
• \. Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythm ia in the past;
• \. Pregnant (or lactating) women;
• \. Patients with severe active infections;
• \. Active infection of hepatitis B virus or hepatitis C virus;
• \. Systemic steroids have used in the 4 weeks before participating in the treatment (except recently or currently using inhaled steroids);
• \. Those who have used any gene therapy products before;
• \. The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
• \. Serum creatinine \> 2.5mg/dl or ALT / AST \> 3 times ULN or bilirubin \> 2.0mg/dl;
• \. Those who suffer from other uncontrolled diseases are not suitable to join the study;
• \. HIV infection;
• \. Any situation that the researchers believe may increase the risk of patients or interfere with the test results.

Sponsors & Collaborators

• Zhejiang University: lead
• Yake Biotechnology Ltd.: collaborator

Study Sites (1)

The first affiliated hospital of medical college of zhejiang university

Hangzhou, Zhejiang, 310000, China

Location

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

Immunotherapy, Adoptive

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Adoptive Transfer; Immunization, Passive; Immunization; Immunotherapy; Immunomodulation; Biological Therapy; Therapeutics; Immunologic Techniques; Investigative Techniques

Study Officials

• He Huang, MD

  Zhejiang University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: April 12, 2023
• First Posted: April 25, 2023
• Study Start: May 15, 2023
• Primary Completion: July 30, 2025
• Study Completion: July 30, 2025
• Last Updated: August 11, 2025

Record last verified: 2025-07

Locations

CN (1)