BCMA/CD3 Bispecific Antibody Treatment for Newly Diagnosed Amyloidosis

Inclusion criteria

• The patient is informed of and voluntarily signs the informed consent form?informed consent form The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later. (ICF).
• Age ≥18 years, regardless of sex.
• Confirmed diagnosis of primary light-chain (AL) amyloidosis, in accordance with the Guidelines for the Diagnosis and Treatment of Systemic Light-chain Amyloidosis (2021 Revision).
• Measurable disease at screening?at screening The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history., defined as:
• Difference between involved and uninvolved free light chains?Difference between involved and uninvolved free light chains The difference between the abnormal ('involved') and normal ('uninvolved') free light chain levels in the blood. Used in some conditions to decide whether disease is measurable and to track response. (dFLC) ≥50 mg/L, or
• Serum involved free light chain ≥50 mg/L with an abnormal κ:λ ratio.
• ECOG performance status?ECOG performance status A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation. ≤2.
• Adequate organ function within 3 days prior to the first dose of the investigational drug?investigational drug A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study., meeting all of the following criteria:
• i. Absolute neutrophil count?Absolute neutrophil count The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level. (ANC) ≥1.0 × 10⁹/L, with no granulocyte colony-stimulating factor (G-CSF) or granulocyte-macrophage colony-stimulating factor (GM-CSF) administration within 7 days, and no pegylated G-CSF administration within 14 days prior to testing; ii. Hemoglobin (Hb) ≥75 g/L, with no whole blood or red blood cell transfusion within 7 days prior to testing; iii. Platelet count ≥70 × 10⁹/L, with no whole blood transfusion, platelet transfusion, or thrombopoietin receptor agonist treatment within 7 days prior to testing; iv. Hepatic function: alanine aminotransferase (ALT) ≤3 × upper limit of normal?upper limit of normal The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value. (ULN), aspartate aminotransferase (AST) ≤3 × ULN, total bilirubin ≤2 × ULN (subjects with Gilbert's syndrome are eligible if direct bilirubin ≤2 × ULN); v. Coagulation: international normalized ratio (INR) or activated partial thromboplastin time (APTT) ≤1.5 × ULN; vi. Renal function: estimated glomerular filtration rate?estimated glomerular filtration rate A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value. (eGFR) ≥20 mL/min/1.73 m², calculated using the CKD-EPI equation.
• Male and female patients of childbearing potential?childbearing potential Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown., and their partners, must agree to use effective contraceptive methods deemed appropriate by the investigator throughout the treatment period and for at least 3 months thereafter.
• Male patients must agree not to donate sperm from the screening period until 90 days after the last dose of the investigational drug.
• The patient must be willing and able to comply with all study procedures and follow-up?follow-up The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes. visits.
• Women not of childbearing potential are eligible for enrollment?enrollment The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.. Women of childbearing potential must have a negative serum or urine β-hCG pregnancy test at screening.
• Note:
• A woman of childbearing potential is defined as a sexually mature woman who has not undergone surgical sterilization (e.g., hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) and has not been postmenopausal for at least 12 consecutive months for reasons other than medical treatment. Women using oral contraceptives or intrauterine devices are considered of childbearing potential. Male subjects (including those who have undergone vasectomy) must agree to use condoms during sexual intercourse with women of childbearing potential and must have no plans to father a child from the time of signing the ICF until 3 months after the last dose of study treatment.