Treatment of Relapsed and/or Refractory AQP4-IgG Seropositive NMOSD by Tandem CAR T Cells Targeting CD19 and CD20

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03605238
• Date of registration: 29/06/2018
• Prospective Registration: Yes
• Primary sponsor: Chinese PLA General Hospital
• Public title: Treatment of Relapsed and/or Refractory AQP4-IgG Seropositive NMOSD by Tandem CAR T Cells Targeting CD19 and CD20
• Scientific title: Clinical Study of CD19/CD20 tanCAR T Cells in Relapsed and/or Refractory AQP4-IgG Seropositive Neuromyelitis Optica Spectrum Disorders (NMOSD)
• Date of first enrolment: August 15, 2018
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT03605238
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

China

Contacts

• Name: Quangang Xu, PhD
• Affiliation: Chinese PLA General Hospital

• Name: Huanfen Zhou, PhD
• Affiliation: Chinese PLA General Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Clinical diagnosis of neuromyelitis optica spectrum disorders (NMOSD) patients.

2. Patients with AQP4-IgG seropositive by cell-based assay.

3. Patients with corticosteroid treatment combined immunosuppressant (azathioprine or
mycophenolate mofetil or rituximab) still recurrence.

4. Clinical evidence of at least 2 relapses in last 12 months or 3 relapses in the last
24 months with at least 1 relapse in the 12 months prior to the Screening.

5. Best corrected visual acuity(BCVA)<20/60.

6. Normal bone marrow reserve function: neutrophils>1 500/mm3, Hemoglobin > 10g/dL,
Platelet count > 100 000/mm3.

7. Normal liver and kidney function: Creatinine < 2.5 mg/dl, ALT (alanine
aminotransferase)/AST (aspartate aminotransferase) < 3x normal, Bilirubin < 2.0 mg/dl.

8. Successful test expansion of tanCART19/20 cells.

9. Adequate venous access for apheresis, and no other contraindications for
leukapheresis.

10. Voluntary informed consent is given.

Exclusion Criteria:

1. Pregnant or lactating women (The safety of this therapy on unborn children is not
known, Female study participants of reproductive potential must have a negative serum
or urine pregnancy test performed within 48 hours before infusion).

2. Any serious, uncontrolled diseases (including, but not limit to, uncontrolled active
infection, active hepatitis B or hepatitis C infection, HIV infection, unstable angina
pectoris, congestive heart failure, serious arrhythmia).

3. Concurrent use of systemic steroids or immunosuppressant in the last two weeks.

4. Feasibility assessment during screening demonstrates < 30% transduction of target
lymphocytes, or insufficient expansion (< 5-fold) in response to CD3/CD137
costimulation.

5. Other patients who are not suitable for CAR-T therapy judged by the biotherapy
physician.



Age minimum: 12 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Neuromyelitis Optica Spectrum Disorder

Intervention(s)

Biological: Corticosteroids & tanCART19/20

Primary Outcome(s)

Occurrence of study related adverse events [Time Frame: From baseline to 12 months after]

Secondary Outcome(s)

Change in Best Corrected Visual Acuity (Log MAR) [Time Frame: Baseline, 12 months]

Annualized relapse rate (ARR) of NMOSD Attacks [Time Frame: Baseline, 12 months]

Change in macular ganglion cell-inner plexiform layers (mGCIPL) [Time Frame: Baseline, 12 months]

Change in Expanded Disability Status Scale (EDDS) Score [Time Frame: Baseline, 12 months]

Change in Flash Visual Evoked Potential (FVEP) [Time Frame: Baseline, 12 months]

Change in peripapillary retinal nerve fibre layer(pRNFL) [Time Frame: Baseline, 12 months]

Secondary ID(s)

CHN-PLAGH-NO-S2018-002-02

Source(s) of Monetary Support

Please refer to primary and secondary sponsors