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| Name | Class |
|---|---|
| Yake Biotechnology Ltd. | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Group | Experimental | Recurrent/Refractory Neuromyelitis Optica |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CAR-T cells injection | Drug | CAR-T cells in the treatment of R/R neuromyelitis optica |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Changes in serum AQP4-IgG titer after infusion | Changes in serum AQP4-IgG titer after infusion | days 7, 14, 21, 28 and 90 |
| Annual recurrence rate (ARR) of NMOSD | Annual recurrence rate (ARR) of NMOSD |
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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
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:
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| Name | Affiliation | Role |
|---|---|---|
| He Huang, MD | Zhejiang University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The first affiliated hospital of medical college of zhejiang university | Hangzhou | Zhejiang | 310000 | China |
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| ID | Term |
|---|---|
| D009471 | Neuromyelitis Optica |
| ID | Term |
|---|---|
| D009188 | Myelitis, Transverse |
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| D016219 | Immunotherapy, Adoptive |
| ID | Term |
|---|---|
| D019264 | Adoptive Transfer |
| D007116 | Immunization, Passive |
| D007114 | Immunization |
| D007167 | Immunotherapy |
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| From admission to the end of the follow-up, up to 2 years |
| Changes in the expanded disability status scale (EDSS) score from baseline | EDSS:0 (normal neurological exam) to 10 (death due to MS),Higher scores indicate greater disability (worse outcome),Score reduction suggests functional improvement | days 7, 14, 21, 28 ,56 and 90 |
| MRI active lesions | Proportion of subjects with ≥1 active lesions at Day 90 | days 90 |
| Changes in optimal corrected vision | Changes in optimal corrected vision (Log MAR) | days 28 and 90 |
| Changes of nerve fiber layer around the retinal papilla(pRNFL) | Change in RNFL by optical coherence tomography over trial | 2 years |
| Changes in the plexiform layer of macular ganglion cells (mGCIPL) | Proportion of subjects with significant mGCIPL thickness changes | 2 years |
| D009902 | Optic Neuritis |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D003711 | Demyelinating Diseases |
| D005128 | Eye Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D056747 |
| Immunomodulation |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D007158 | Immunologic Techniques |
| D008919 | Investigative Techniques |