Systemic light chain amyloidosis
Conditions
Interventions
Research group:Aponermin (Apo) combined with CyBorD
Sponsors
Tianjin Medical University General Hospital
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1.Be aware of and voluntarily sign the Informed Consent Form (ICF); 2.Age >=18 years old; 3.Clearly diagnosed primary light chain amyloidosis: Refer to the standards of the Guidelines for the Diagnosis and Treatment of Primary Light Chain amyloidosis (revised in 2021); 4.Measurable diseases at the time of screening: serum free light chain difference (dFLC, difference between affected light chain and unaffected light chain) > 50 mg/L; 5.Expected survival period > 12 weeks; 6.No active infection; 7.HBV-DNA negative, HCV-RNA negative, HIV negative; 8.Non-childbearing women meet the inclusion requirements; Female patients of childbearing age must have a negative serum (ß -human chorionic gonadotropin) or urine pregnancy test at screening.
Exclusion criteria
Exclusion criteria: 1.Non-Al amyloidosis: including hereditary amyloidosis and other non-Al amyloidosis; 2.Combined with multiple myeloma: Refer to the Chinese Guidelines for the Diagnosis and Treatment of Multiple Myeloma (revised in 2022); 3.Regardless of whether the patient is currently undergoing drug treatment or not, at the time of screening, there is peripheral neuropathy of grade >2 or painful neuropathy of grade >=2; 4.It is known that there is intolerance, allergy or contraindications to any of the active ingredients of Aponermin or CyBorD; 5.According to the improved Mayo2004 staging system, the stage is IIIb. 6.Patients with unstable or active cardiovascular and cerebrovascular system diseases, meeting any of the following conditions: (1)Unstable angina pectoris, myocardial infarction or coronary artery reconstruction surgery occurred within 180 days before the first administration. ; (2)Congestive heart failure (CHF)grade >=3 as defined by the New York Heart Association (NYHA); (3)Second-degree and above atrioventricular block that cannot be controlled by conventional treatment; Persistent or recurrent non-persistent ventricular tachycardia; (4)A history of stroke or intracranial hemorrhage within 12 months prior to screening; (5)There were severe thrombotic events before treatment; 7.Known active human immunodeficiency virus (HIV.infection or HIV seropositive; 8.Severe pulmonary dysfunction (pulmonary carbon monoxide diffusion capacity DLCO 2.5 times the upper limit of normal, or total bilirubin > 1.5mg/dL, or creatinine clearance rate < 40mL/min (< 30mL/min in patients with AL involving the kidneys); 11.Severe blood abnormalities, specifically: absolute neutrophil count < 1×10^9/L, or hemoglobin < 7g/dL, or platelet count < 75×10^9/L; 12.Pregnant or lactating women; 13.Organ transplantation has been performed previously; 14.Patients have undergone major surgeries (for example, requiring general anesthesia.within 2 weeks before enrollment, or will not fully recover from the surgery, or have surgical arrangements during the planned participation in the study. Kyphoplasty or vertebroplasty is not regarded as a major operation. Note: Patients who are planned to undergo surgical operations under local anesthesia can participate in the study; 15.Have received attenuated live vaccines within 4 weeks before the administration of the first study drug; 16.According to the determination of the researcher, any active severe mental or psychological illness, medical illness or other symptoms/conditions that may affect treatment, compliance or the ability to provide informed consent; 17.There are contraindications to any required concomitant medications or supportive treatments; 18.There are any diseases or complications that may interfere with the research process; 19.The patient is unwilling
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| ORR; | — |
Secondary
| Measure | Time frame |
|---|---|
| Remission rate of affected organs;PFS;TTR;Optimal hematological remission depth;OS;Hematological and non-hematological toxicity; | — |
Countries
China
Contacts
Public ContactFu Rong
Tianjin Medical University General Hospital
Outcome results
None listed