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

NCT ID: NCT05828212

Last Updated: 2025-08-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-05-15
• Study Completion Date: 2025-07-30

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment Group

Recurrent/Refractory Neuromyelitis Optica

Group Type: EXPERIMENTAL

CAR-T cells injection

Intervention Type: DRUG

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

Interventions

CAR-T cells injection

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

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Yake Biotechnology Ltd.

INDUSTRY

Sponsor Role: collaborator

Zhejiang University

OTHER

Sponsor Role: lead

Responsible Party

He Huang

Clinical Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

He Huang, MD

Role: PRINCIPAL_INVESTIGATOR

Zhejiang University

Locations

The first affiliated hospital of medical college of zhejiang university

Hangzhou, Zhejiang, China

Countries

China

Other Identifiers

TXB2023005

Identifier Type: -

Identifier Source: org_study_id