Autoimmune Diseases, Scleroderma
Conditions
Keywords
Scleroderma, CD19 CAR T-cell therapy, BCMA CAR T-cell therapy
Brief summary
A Study of CD19/BCMA Chimeric Antigen Receptor T Cells Therapy for Patients With Refractory Scleroderma
Detailed description
Autoimmune diseases only show local pathological damage, but more often systemic lesions. If not diagnosed and treated in time or poorly controlled, a risk of disability or even death as the course of the disease progresses. Studies have shown that B cells can present their own antigens to autoimmune T cells to promote the release of inflammatory factors, or they can differentiate into plasma cells to release autoantibodies, and play an important role in the occurrence and progression of autoimmune diseases. In recent years, it has become a major research focus to deplete B cells in patients or inhibit B cell function. This research focuses on CAR-T cells killing B cells. This fully reflects the application prospects of CAR-T cells in autoimmune diseases. Based on the current research progress, our center intends to conduct research on the safety and effectiveness of CD19/BCMA CAR-T cells in the treatment of refractory scleroderma
Interventions
Drug: CD19/BCMA CAR T-cells Each subject receive CD19/BCMA CAR T-cells by intravenous infusion Other Name: CD19/BCMA CAR T-cells injection
Sponsors
Yake Biotechnology Ltd.
Zhejiang University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
1. Scleroderma with positive CD19/BCMA expression , and the conventional treatment is not effective and (or) no effective treatment 2. Estimated survival time\> 12 weeks; 3. 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; 4. 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 CAR T-cells infusion | Adverse events assessed according to NCI-CTCAE v5.0 criteria |
| Incidence of treatment-emergent adverse events (TEAEs) | Up to 90 days after CD19/BCMA CAR T-cells infusion | Incidence of treatment-emergent adverse events \[Safety and Tolerability\] |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Disease control rate, DCR | From Day 28 CD19/BCMA CAR-T infusion up to 2 years | The percentage of patients with remission and stable disease after treatment in the total evaluable cases. |
| Duration of remission, DOR | 24 months 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 |
| Concentration of CAR-T cells | From admission to the end of the follow-up, up to 2 years | In peripheral blood |
| 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 |
| Progression-free survival, PFS | 24 months post CD19/BCMA CAR-Tcells infusion | The time from cell reinfusion to the first assessment of disease progression or death from any cause |
| Objective Response Rate, ORR | In 3 months of CD19/BCMA CAR-T cell infusion | Proportion of subjects with complete or partial remission |
Countries
China
Contacts
Primary ContactHe Huang, PhD
Backup ContactYongxian Hu, PhD
Outcome results
None listed