Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis
RATIONALE: Giving melphalan and bortezomib before and after a stem cell transplant stops the growth of abnormal cells by stopping them from dividing or killing them. Giving colony-stimulating factors and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy and monoclonal antibody therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.
PURPOSE: This phase II trial is studying how well giving melphalan together with bortezomib followed by stem cell transplant works in treating patients with primary systemic amyloidosis.
Procedure: Stem Cell Infusion
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Trial of High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis|
- Number of Participants With Hematologic Response [ Time Frame: one year ]
complete and partial hematologic response defined as: Complete response: absence of detectable monoclonal protein in serum and urine, and bone marrow biopsy <5% plasma cells with no clonal predominance of kappa or lambda isotype.
Partial response: any one of the following
- For patients with detectable and quantifiable marrow plasmacytosis, a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells.
- For patients with a detectable monoclonal peak on serum protein electropheresis or urine protein electropheresis, a reduction in the peak height of 50% or more.
- For patients with quantifiable urinary kappa or lambda chain concentration, a reduction in daily light chain excretion (concentration x 24-hr urine volume).
- Number of Participants Surviving at 100 Days From Transplant [ Time Frame: 100 Days from transplant date ]
- Number of Participants Surviving at 1 Year [ Time Frame: one year from transplant ]
- Number of Participants Surviving at 2 Years [ Time Frame: 2 years from transplant ]
|Study Start Date:||June 2008|
|Study Completion Date:||November 2014|
|Primary Completion Date:||December 2012 (Final data collection date for primary outcome measure)|
Experimental: Stem Cell Transplant with Bortezomib and Melphalan
Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
16 mcg/kg daily beginning 3 days before stem cell collection through day before final stem cell collection
Other Name: Growth-Colony Stimulating Factor, neupogenDrug: bortezomib
1.0 mg/m2/dose D -6, D-3, D +1, D + 4
Other Name: velcadeDrug: melphalan
100 mg/m2/dose D -2, D -1
Other Name: alkeranProcedure: Stem Cell Infusion
infusion of previously collected autologous stem cells
- To determine if hematologic responses to high-dose melphalan and autologous stem cell transplantation increase with addition of bortezomib in the conditioning regimen in patients with primary systemic amyloidosis.
- Autologous stem cell mobilization and collection: Patients receive filgrastim to mobilize stem cells, which are then collected.
- Conditioning regimen: Patients receive bortezomib intravenously on days -6, -3, 1, and 4 and oral high-dose melphalan on days -2 and -1.
- Stem cell transplantation: Patients undergo autologous stem cell transplantation on day 0.
After completion of study therapy, patients are followed every 6 months for 1 year and annually thereafter.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00790647
|United States, Massachusetts|
|Boston University Cancer Research Center|
|Boston, Massachusetts, United States, 02118|
|Principal Investigator:||Vaishali Sanchorawala, MD||Boston Medical Center|