Patients with light chain (AL) amyloidosis who have advanced cardiac damage are at risk of premature mortality. There is ongoing unmet need for effective therapies to rapidly induce deep hematologic response and decrease the early death rate. Lately, trials of daratumumab in newly-diagnosed and relapsed/refractory AL amyloidosis have shown dramatic response rates. However, the benefits of upfront daratumumab in stage III AL patients, especially stage IIIb patients, have not yet been demonstrated definitely in prospective studies. Therefore, we designed a phase II, single arm clinical trial to investigate the efficacy and safety of co-administration of daratumumab with bortezomib and dexamethasone (BD) regimen in treatment-naïve patients with Mayo 04 stage III AL amyloidosis. We planned to enroll 40 patients, who would receive daratumumab and BD treatment for a total duration of 12 months. The primary endpoint is complete response and very good partial response at 3 months after treatment initiation. Secondary endpoints include overall survival, organ response and adverse events.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
40
16mg/kg, QW Cycles 1-2 (28 days/cycle), Q2W Cycles 3-6, and Q4W thereafter for up to 1 year
1.3mg/m2 of subcutaneous bortezomib on days 1, 8, 15 and 22 of a 28-day cycle for 6 cycles
20mg of dexamethasone on days 1, 8, 15 and 22 of a 28-day cycle for 6 cycles
Peking Union Medical College Hospital
Beijing, China
Hematologic very good partial response or better at 3 months after treatment initiation
Very good partial response or better is defined as complete response or very good partial response. Complete response: normalization of free light chain levels and ratio with negative serum and urine immunofixation electrophoresis. Very good partial response: difference between involved and uninvolved free light chains (dFLC) less than 40 mg/L
Time frame: 3 months
Overall survival
Time frame: 2 years
major organ deterioration progression-free survival
Time frame: 2 years
Time to next treatment
Time frame: 2 years
Mortality within 1 month from treatment initiation
Time frame: 1 month
Mortality within 3 months from treatment initiation
Time frame: 3 months
Mortality within 6 months from treatment initiation
Time frame: 6 months
Hematologic very good partial response or better at 1 month after treatment initiation
Time frame: 1 month
Hematologic very good partial response or better at 6 months after treatment initiation
Time frame: 6 months
Hematologic very good partial response or better at 12 months after treatment initiation
Time frame: 12 months
Stringent dFLC response
dFLC declined to less than 10 mg/L
Time frame: 1 year
Time to hematologic response
Time frame: 1 year
Organ response at 3 months after treatment initiation
Time frame: 3 months
Organ response at 6 months after treatment initiation
Time frame: 6 months
Organ response at 12 months after treatment initiation
Time frame: 12 months
Time to cardiac response
Time frame: 1 year
Time to liver response
Time frame: 1 year
Time to renal response
Time frame: 1 year
Adverse events
Adverse events are collected until 30 days after last dose of treatment
Time frame: treatment initiation to 30 days after last dose of treatment
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