Amyloidosis, Autoimmune Hemolytic Anemia, POEMS Syndrome, Vasculitis
Conditions
Keywords
CD19 CAR-T, BCMA CAR-T, POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, Vasculitis
Brief summary
A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
Detailed description
POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis and other diseases may only show local pathological damage or systemic lesions. If they are not diagnosed and treated in time or poorly controlled, they will progress as the course of the disease progresses. Risk of disability or even death.
Interventions
BIOLOGICALCD19/BCMA CAR T-cells
Each subject receive CD19/BCMA CAR T-cells by intravenous infusion
Sponsors
Yake Biotechnology Ltd.
Zhejiang University
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* 1\. Diagnosed with POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis, and the curative effect of conventional hormones, radiotherapy and chemotherapy, protease inhibitors is not good and (or) no effective treatment means. 2\. After glucocorticoids, cyclophosphamide or methotrexate treatments there are still relapsed and refractory diseases, or clearly show intolerance/toxicity to these drugs. 3\. Estimated survival time\> 12 weeks; 4. Patients had a negative urine pregnancy test before the start of administration and agreed to take effective contraceptive measures during the test period until the last follow-up; 5. Patients or their legal guardians volunteer to participate in the study and sign the informed consent.
Exclusion criteria
* Subjects with any of the following
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Dose-limiting toxicity (DLT) | Baseline up to 28 days after CD19/BCMA targeted CAR T-cells infusion | Adverse events assessed according to NCI-CTCAE v5.0 criteria |
| Incidence of treatment-emergent adverse events (TEAEs) | Up to 2 years after CD19/BCMA targeted CAR T-cells infusion | Incidence of treatment-emergent adverse events \[Safety and Tolerability\] |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Best overall response, BOR | At ≤3 month | Assessment of ORR at ≤3 month |
| Titer of auto-antibody Titer of auto-antibody titer of auto-antibody | Up to 2 years after CD19/BCMA targeted CAR T-cells infusion | In peripheral blood and bone marrow |
| Duration of remission, DOR | 2 years post CD19/BCMA CAR-T cells infusion | The time from the first assessment of remission or partial remission of the disease to the first assessment of disease progression or death from any cause |
| Overall survival (OS) | From CD19/BCMA CAR-T infusion to death，up to 2 years | The time from the cell reinfusion to death due to any cause |
| Overall response rate (ORR) | Up to 2 years after CD19/BCMA targeted CAR T-cells infusion | Proportion of subjects with complete or partial remission |
Countries
China
Contacts
Primary ContactHe Huang, PhD
Backup ContactYongxian Hu, PhD
Outcome results
None listed