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

Zhejiang University

Status and phase

Completed

Phase 1

Conditions

Neuromyelitis Optica

Treatments

Drug: CAR-T cells injection

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT05828212

TXB2023005

Details and patient eligibility

About

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.

Enrollment

9 patients

Sex

All

Ages

18 to 60 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

1. Age 18-60 and gender unlimited;
1. NMOSD diagnosed based on the 2015 NMOSD International Consensus Diagnostic Criteria;
1. Diagnostic criteria for AQP4-IgG positive NMOSD Diagnostic criteria for AQP4-IgG positive NMOSD
2. At least 1 core clinical feature
3. Using reliable methods to detect positive AQP4-IgG (CBA method)
4. Exclude other diagnoses. Core clinical features

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
1. Corticosteroids combined with immunosuppressants (azathioprine or mycophenolate mofetil or rituximab) still relapse after treatment；
1. 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；
1. The estimated survival time is more than 12 weeks;
1. 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;
1. Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythm ia in the past;
1. Pregnant (or lactating) women;
1. Patients with severe active infections;
1. Active infection of hepatitis B virus or hepatitis C virus;
1. Systemic steroids have used in the 4 weeks before participating in the treatment (except recently or currently using inhaled steroids);
1. Those who have used any gene therapy products before;
1. The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
1. Serum creatinine > 2.5mg/dl or ALT / AST > 3 times ULN or bilirubin > 2.0mg/dl;
1. Those who suffer from other uncontrolled diseases are not suitable to join the study;
1. HIV infection;
1. Any situation that the researchers believe may increase the risk of patients or interfere with the test results.