This is a phase 1/2 open-label, dose-escalation study investigating single-agent therapy with VELCADE in patients with previously treated systemic AL-amyloidosis who require further treatment.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
70
Once weekly at: 0.7, 1.0, 1.3 or 1.6 mg/m2 Or Twice-weekly at: 0.7, 1.0, or 1.3 mg/m2
Cedars-Sinai Medical Center, Samuel Oschin Comprehensive Cancer Institute
Los Angeles, California, United States
Winship Cancer Center - Emory Clinic School of Medicine
Atlanta, Georgia, United States
Boston Medical Center
Boston, Massachusetts, United States
MSKCC
New York, New York, United States
Maximum Tolerated Dose
Maximum Tolerated Dose (MTD) was defined as the highest dose level that has 0/1 out of 6 patients experiences Dose Limited Toxicity (DLT). MTD is defined separately for QW and BIQ dose cohorts. DLT was defined as adverse events occurring during Cycle 1 and: (1) related to VELCADE, (2) Grade 4 thrombocytopenia or neutropenia, (3) Grade 3 or higher nonhematologic toxicity.
Time frame: 5 weeks in once weekly (QW) dose cohorts and 3 weeks in twice weekly (BIW) dose cohorts
Subjects With Treatment Emergent Adverse Events
Treatment emergent adverse events observed during outcome measure time frame
Time frame: from first study-related procedure to 30 days after last dose of study medication
Subjects With Serious Treatment Emergent Adverse Events
Serious treatment emergent adverse events observed during outcome measure time frame
Time frame: from first study-related procedure to 30 days after last dose of study medication
Subjects Grade 3/4/5 Treatment Emergent Adverse Events
Grade 3/4/5 treatment emergent adverse events observed during outcome measure time frame. Grade is determined according to Common Terminology Criteria for Adverse Event (CTCAE) Version 3.0.
Time frame: from first study-related procedure to 30 days after last dose of study medication
Subjects With Treatment Emergent Adverse Events Leading to Treatment Termination
Treatment emergent adverse events observed during outcome measure time frame leading to treatment termination
Time frame: from first study-related procedure to 30 days after last dose of study medication
Best Confirmed Hematologic Responders
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Hematologic response was determined by the investigator per the response criteria for immunoglobulin light chain amyloidosis by Gertz (2005). It include Complete and Partial Responders (CR+PR). CR requires serum and urine negative for a monoclonal protein by immunofixation and free light chain ratio normal. PR requires: 1. reduction in quantitative serum M-protein by 50% if baseline value is at least 0.5 g/dL, 2. if light chain is detected in the urine (with a consistent peak and \>100 mg/ 24 hours), then 50% reduction is required, 3. if free light chain \>10 mg/dL, reduction by 50% is required.
Time frame: from first dose of study medication to end of study visit