This phase I trial studies the side effects and best dose of pomalidomide and bortezomib when given together with dexamethasone in treating patients with amyloid light-chain amyloidosis or light chain deposition disease. Biological therapies, such as pomalidomide, may stimulate the immune system in different ways and stop abnormal cells from growing. Bortezomib may stop the growth of abnormal cells by blocking some of the enzymes needed for cell growth. Giving pomalidomide and bortezomib together with dexamethasone may be an effective treatment for amyloid light-chain amyloidosis or light chain deposition disease
PRIMARY OBJECTIVES: I. Establish the maximum tolerated dose (MTD) of the combination of pomalidomide, bortezomib, and dexamethasone (PVD) to take forward in a subsequent phase 2 study. SECONDARY OBJECTIVES: I. Obtain a preliminary assessment of efficacy of PVD regimen as initial treatment of amyloid light-chain (AL) or light chain deposition disease (LCDD). OUTLINE: This is a dose-escalation study of pomalidomide and bortezomib. Patients receive pomalidomide orally (PO) on days 1-21; bortezomib intravenously (IV) on days 1, 8, and 15; and dexamethasone PO on days 1, 8, 15, and 22. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at least every 3 months.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
18
Colorado Blood Cancer Institute
Denver, Colorado, United States
Boston University School of Medicine
Boston, Massachusetts, United States
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, United States
Duke University Medical Center
Durham, North Carolina, United States
Princess Margaret Cancer Centre
Toronto, Ontario, Canada
Maximum tolerated dose defined as the dose level before 2 of 6 patients experience dose-limiting toxicity (DLT) using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
Time frame: 28 days
Complete hematologic response rate (hCR)
Will be estimated and reported with 95% confidence intervals.
Time frame: Up to 28 days
Overall hematologic response rate (partial response [PR] + complete response [CR])
Time frame: Up to 28 days
Organ response rates (heart, liver, kidney)
Organ responses will be tabulated and reported for all patients with cardiac, renal, hepatic, and/or neurologic involvement by amyloidosis.
Time frame: Up to 28 days
Overall survival
Will be estimated and reported with 95% confidence interval.
Time frame: From date of registration to date of death, assessed up to 28 days
Progression free survival
Will be estimated and reported with 95% confidence interval.
Time frame: Up to 28 days
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