Light Chain Amyloidosis
Conditions
Brief summary
This study is a multicenter, open label, fixed dose exploratory clinical trial with an expected enrollment of 30 subjects. The main objective is to evaluate the safety of targeted BCMA autologous CART cell injection in the treatment of recurrent or refractory light chain amyloidosis in subjects, preliminarily verify the effectiveness of targeted BCMA autologous CART cell injection in the treatment of recurrent or refractory light chain amyloidosis in subjects, and explore the pharmacokinetic, pharmacodynamic, and immunogenic characteristics of targeted BCMA autologous CART cell injection after reinfusion.
Interventions
GENETICBCMA CART
The cell product used in this study is a targeted BCMA autologous CART cell injection. Research drug name: Targeted BCMA Autologous CART Cell Injection Drug code: BJBR-BCMA-006 Administration method: single intravenous injection
Sponsors
Beijing Boren Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
\- Participants must meet all inclusion criteria, and only those who do not meet any
Exclusion criteria
can be enrolled 1. Participants must personally sign the informed consent form approved by the ethics committee in writing before the start of the study; 2. The age of the subject is ≥ 18 years old; 3. Diagnosed with light chain amyloidosis through pathological examination; 4. Subjects with recurrent/refractory light chain amyloidosis who have previously received second-line or higher treatment; 5. dFLC \> 50mg/L 6. Expected survival period ≥ 12 weeks; 7. ECOG score ≤ 2 points; 8. Female subjects with fertility should agree to take effective contraceptive measures from the date of signing the informed consent form until 365 days after reinfusion. Effective contraceptive measures are defined as abstinence or using contraceptive methods with an annual failure rate of less than 1% as specified in the plan; 9. Prior to enrollment, participants must have appropriate organ function and meet all of the following test results: 9.1 Absolute neutrophil count ≥ 1.0 × 109/L \[allowed to use granulocyte colony-stimulating factor (G-CSF) support\]; 9.2 Platelet count ≥ 50 × 109/L; 9.3 Hemoglobin ≥ 8 g/dl; 9.4 Bilirubin value ≤ 1.5 x upper limit of normal (ULN); 9.5 ALT or AST ≤ 2.5 times the upper limit of normal (ULN) (with liver involvement ≤ 5 times the upper limit of normal); 9.6 Mayo 2004 Stage I-IIIa participants; 9.7 Stable coagulation function: INR ≤ 1.5, APTT ≤ 1.2 x upper limit of normal (ULN); Basic blood oxygen saturation is greater than 92% in indoor natural air environment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Frequency Of AES, SAES | 0 day to 24 months after treatment |
| Dose Limiting Toxicity | 0~28 day after treatment |
Countries
China
Contacts
Primary Contactyajing zhang, MD/PhD
Outcome results
None listed