This is a prospective, single-arm, single-center clinical study designed to evaluate the efficacy and safety of low-dose BCMA/CD3 bispecific antibody (CM336) in patients newly diagnosed with systemic light chain (AL) amyloidosis.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
21
CM336 is a bispecific T-cell engager targeting B-cell maturation antigen (BCMA) and CD3. In this study, CM336 is administered subcutaneously with a step-up dosing strategy in Cycle 1 (3 mg Day 1, 20 mg Day 4, 40 mg Day 8 and onwards weekly). Patients who achieve ≥VGPR by Cycle 4 may switch to 80 mg every two weeks from Cycle 5. The total treatment duration is up to 12 cycles (28 days per cycle), with follow-up for safety and efficacy endpoints including hematologic and organ response.
Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences
Tianjin, China
RECRUITINGRate of Hematologic Very Good Partial Response (VGPR) or Better
Proportion of participants achieving a hematologic response of VGPR or better (≥VGPR) after 4 treatment cycles of anti-BCMA/CD3 bispecific antibody (CM336), assessed using consensus criteria for AL amyloidosis hematologic response.
Time frame: From the start of treatment (Day 0), at the end of Cycle 4 (each cycle is 28 days), at the end of Cycle 8 (each cycle is 28 days), and at the end of Cycle 12 (each cycle is 28 days)
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Safety will be assessed by monitoring the incidence, nature, and severity of treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs) such as cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), graded according to NCI CTCAE v5.0 and ASTCT criteria. Dose interruptions, modifications, or discontinuations due to toxicity will also be recorded.
Time frame: From the first dose through 30 days after the last dose, up to approximately 24 months.
Time to First Hematologic Response (TTR)
Time frame: From the first dose until the best hematologic response (≥PR) is achieved, assessed up to approximately 24 months.
Best Hematologic Response Achieved
The deepest hematologic response (e.g., PR, VGPR, CR, or sCR) observed at any time during the treatment period.
Time frame: From the first dose until the best hematologic response (≥PR) is achieved, assessed up to approximately 24 months.
Duration of Hematologic Response (DOR)
Time from the first documented hematologic response to disease progression or death, whichever occurs first.
Time frame: From the date of first documented hematologic response to the date of disease progression or death, whichever occurs first, up to approximately 24 months.
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Overall Response Rate (ORR)
Time frame: The overall response rate (ORR) was evaluated at the end of cycle 4, 6, and 12 (28 days per cycle).
Progression-Free Survival (PFS)
Time frame: From the first dose to progression from any cause, up to approximately 36 months.
Overall Survival (OS)
Time frame: From the first dose to death from any cause, up to approximately 36 months.
Minimal Residual Disease (MRD) Negativity Rate
Time frame: At the time of achieving hematologic complete response, up to approximately 24 months.
Standardized uptake value (SUV)
Standardized uptake value (SUV) of 18F-92/AV45/TPZA/FT8, 11C-PIB for each target lesion of subject.
Time frame: At baseline (prior to first dose), at the end of Cycle 6, and Cycle 12 (each cycle = 28 days)