First-line Pomalidomide, Bortezomib, and Dexamethasone For AL Amyloidosis or LCDD
Light Chain Deposition Disease
Primary Systemic Amyloidosis
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase I Study of Pomalidomide, Bortezomib, and Dexamethasone (PVD) as First-Line Treatment of AL Amyloidosis or Light Chain Deposition Disease|
- 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 ] [ Designated as safety issue: Yes ]
- Complete hematologic response rate (hCR) [ Time Frame: Up to 28 days ] [ Designated as safety issue: No ]Will be estimated and reported with 95% confidence intervals.
- Overall hematologic response rate (partial response [PR] + complete response [CR]) [ Time Frame: Up to 28 days ] [ Designated as safety issue: No ]
- Organ response rates (heart, liver, kidney) [ Time Frame: Up to 28 days ] [ Designated as safety issue: No ]Organ responses will be tabulated and reported for all patients with cardiac, renal, hepatic, and/or neurologic involvement by amyloidosis.
- Overall survival [ Time Frame: From date of registration to date of death, assessed up to 28 days ] [ Designated as safety issue: No ]Will be estimated and reported with 95% confidence interval.
- Progression free survival [ Time Frame: Up to 28 days ] [ Designated as safety issue: No ]Will be estimated and reported with 95% confidence interval.
|Study Start Date:||March 2013|
|Estimated Study Completion Date:||June 2016|
|Estimated Primary Completion Date:||June 2016 (Final data collection date for primary outcome measure)|
Experimental: Treatment (pomalidomide, bortezomib, and dexamethasone)
Patients receive pomalidomide PO on days 1-21; bortezomib 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.
Other Name: CC-4047Drug: bortezomib
Other Names:Drug: dexamethasone
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.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01728259
|United States, Colorado|
|Colorado Blood Cancer Institute|
|Denver, Colorado, United States, 80218|
|United States, Massachusetts|
|Boston University School of Medicine|
|Boston, Massachusetts, United States, 02118-2393|
|United States, Michigan|
|Barbara Ann Karmanos Cancer Institute|
|Detroit, Michigan, United States, 48201|
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27710|
|Princess Margaret Cancer Centre|
|Toronto, Ontario, Canada, M5G 2M9|
|Principal Investigator:||Jeffrey Zonder||Barbara Ann Karmanos Cancer Institute|