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

Sponsor
Zhejiang University (Other)
Overall Status
Recruiting
CT.gov ID
NCT05828212
Collaborator
(none)
9
Enrollment
1
Location
1
Arm
36
Anticipated Duration (Months)
0.2
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: CD19 CAR-T cells injection
 Phase 1

Study Design

Study Type:
Interventional
Anticipated Enrollment :
9 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Study on the Safety and Efficacy of Chimeric Antigen Receptor T Cells in the Treatment of Recurrent/Refractory Neuromyelitis Optica
Anticipated Study Start Date :
Apr 30, 2023
Anticipated Primary Completion Date :
Apr 30, 2026
Anticipated Study Completion Date :
Apr 30, 2026

Arms and Interventions

Arm Intervention/Treatment
Experimental: Treatment Group

Recurrent/Refractory Neuromyelitis Optica

Drug: CD19 CAR-T cells injection
CD19 CAR-T cells in the treatment of R/R neuromyelitis optica

Outcome Measures

Primary Outcome Measures

  1. Dose limited toxicity (DLT) [From date of initial treatment to Day 28 post CD19 CAR-T infusion.]

    Dose limited toxicity

  2. AE and SAE [From admission to the end of the follow-up, up to 2 years]

    Adverse event and serious adverse event

  3. Maximum tolerable dose [From date of initial treatment to Day 28 post CD19 CAR-T infusion.]

    Maximum tolerable dose

Secondary Outcome Measures

  1. Changes in serum AQP4-IgG titer after infusion [days 7, 14, 21, 28 and 90]

    Changes in serum AQP4-IgG titer after infusion

  2. Annual recurrence rate (ARR) of NMOSD [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 [days 7, 14, 21, 28 ,56 and 90]

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

  4. MRI active lesions [days 90]

    MRI active lesions

  5. Changes in optimal corrected vision [days 28 and 90]

    Changes in optimal corrected vision (Log MAR)

  6. Changes of nerve fiber layer around the retinal papilla(pRNFL) [2 years]

    Change in RNFL by optical coherence tomography over trial

  7. Changes in the plexiform layer of macular ganglion cells (mGCIPL) [2 years]

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

  8. Changes in Flash Visual Evoked Potential (FVEP) [2 years]

    Changes in Flash Visual Evoked Potential (FVEP)

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 60 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

  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

  4. ＯＮ

  5. Acute myelitis

  6. Posterior region syndrome, unexplained paroxysmal hiccup, nausea, and vomiting

  7. Other brainstem syndromes

  8. Symptomatic episodic sleeping sickness, diencephalic syndrome, brain MRI with NMOSD characteristic diencephalic lesions

  9. 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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 The first affiliated hospital of medical college of zhejiang university Hangzhou Zhejiang China 310000

Sponsors and Collaborators

  • Zhejiang University

Investigators

  • Principal Investigator: He Huang, MD, Zhejiang University

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
He Huang, Clinical Professor, Zhejiang University
ClinicalTrials.gov Identifier:
NCT05828212
Other Study ID Numbers:
  • TXB2023005
First Posted:
Apr 25, 2023
Last Update Posted:
Apr 25, 2023
Last Verified:
Apr 1, 2023
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Neuromyelitis Optica

Study Results

No Results Posted as of Apr 25, 2023