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Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis

Phase 2
Completed
Conditions
Multiple Myeloma
Interventions
Biological: filgrastim
Procedure: Stem Cell Infusion
Registration Number
NCT00790647
Lead Sponsor
Boston Medical Center
Brief Summary

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.

Detailed Description

OBJECTIVES:

* 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.

OUTLINE:

* 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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
10
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Stem Cell Transplant with Bortezomib and MelphalanStem Cell InfusionMobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
Stem Cell Transplant with Bortezomib and MelphalanfilgrastimMobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
Stem Cell Transplant with Bortezomib and MelphalanbortezomibMobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
Stem Cell Transplant with Bortezomib and MelphalanmelphalanMobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
Primary Outcome Measures
NameTimeMethod
Number of Participants With Hematologic Responseone 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

1. 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.

2. 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.

3. For patients with quantifiable urinary kappa or lambda chain concentration, a reduction in daily light chain excretion (concentration x 24-hr urine volume).

Secondary Outcome Measures
NameTimeMethod
Number of Participants Surviving at 100 Days From Transplant100 Days from transplant date
Number of Participants Surviving at 1 Yearone year from transplant
Number of Participants Surviving at 2 Years2 years from transplant

Trial Locations

Locations (1)

Boston University Cancer Research Center

🇺🇸

Boston, Massachusetts, United States

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