Actively Recruiting
Exploratory Clinical Study on the Safety and Efficacy of Targeted BCMA Autologous CART Cell Injection in Subjects With Recurrent/Refractory Light Chain Amyloidosis
Led by Beijing Boren Hospital · Updated on 2025-05-21
30
Participants Needed
1
Research Sites
136 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Exploratory Clinical Study on the Safety and Efficacy of Targeted BCMA Autologous CART Cell Injection in Subjects With Recurrent/Refractory Light Chain Amyloidosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be 18 years or older
- Signed informed consent form approved by the ethics committee
- Diagnosed with light chain amyloidosis by pathological examination
- Have recurrent or refractory light chain amyloidosis after second-line or higher treatment
- dFLC greater than 50 mg/L
- Expected survival of at least 12 weeks
- ECOG performance status score of 2 or less
- Females with reproductive potential must agree to use effective contraception from consent until 365 days after reinfusion
- Adequate organ function including neutrophil count ≥1.0 x 10^9/L (G-CSF support allowed), platelet count ≥50 x 10^9/L, hemoglobin ≥8 g/dl
- Bilirubin ≤1.5 times upper limit of normal (ULN)
- ALT or AST ≤2.5 times ULN (≤5 times ULN with liver involvement)
- Mayo 2004 Stage I to IIIa
- Stable coagulation function: INR ≤1.5, APTT ≤1.2 times ULN
- Basic blood oxygen saturation greater than 92% in room air
You will not qualify if you...
- Prior gene therapy
- Received live vaccines within 4 weeks before enrollment
- Received other investigational drug treatments within 12 weeks before collection
- Central nervous system involvement or complete intestinal obstruction
- Moderate to severe pleural or peritoneal effusion requiring continuous drainage
- Active malignancies within 5 years except cured basal/squamous cell carcinoma or carcinoma in situ
- Positive hepatitis B surface antigen with abnormal HBV DNA or positive hepatitis C antibodies with RNA, HIV positive, CMV DNA positive, or positive syphilis test
- Uncontrolled active infections (except mild urinary, reproductive, or upper respiratory infections)
- Severe heart disease including unstable angina, recent myocardial infarction, congestive heart failure NYHA class III or higher, significant arrhythmias, or thickened heart walls
- Hypertension not controlled by medication
- Unresolved treatment toxicities above grade 1 except hair loss or minor lab abnormalities
- Major surgery within 2 weeks before enrollment or planned surgery during reinfusion wait or within 12 weeks after treatment (except minor local anesthesia surgery)
- Solid organ transplant recipients
- Pregnant or breastfeeding women
- History of central nervous system disorders or consciousness disorders
- Unstable systemic diseases requiring medication, including severe liver, kidney, or metabolic conditions
- Known life-threatening allergies or intolerance to cellular products
- Diagnosed bleeding or thrombosis disorders or on thrombolytic or anticoagulant therapy
- Any other conditions deemed unsuitable by researchers
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Beijing Gobroad Brond Hospital
Beijing, Beijing Municipality, China, 100070
Actively Recruiting
Research Team
Y
yajing zhang, MD/PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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