Standard treatment for AL Amyloidosis is high-dose melphalan and stem cell transplant. This study will compare the safety and effectiveness of standard treatment with high-dose melphalan and stem cell transplant, compared with investigational bortezomib when used in combination with standard treatment with high-dose melphalan and stem cell transplant for AL amyloidosis.
This study seeks to enroll patients with AL amyloidosis who have been recommended for standard treatment with high-dose melphalan and stem cell transplant. Standard treatment for this disease is high-dose melphalan and stem cell transplant. The purpose of this study is to compare the safety and effectiveness of standard treatment with high-dose melphalan and stem cell transplant, compared with investigational bortezomib when used in combination with standard treatment with high-dose melphalan and stem cell transplant for AL amyloidosis. Patients enrolled in this study will receive either standard treatment with high-dose melphalan and stem cell transplant, or investigational bortezomib used in combination with standard treatment with high-dose melphalan and stem cell transplant.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
3
Conditioning Regimen: Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1
Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0
granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days
Boston Medical Center
Boston, Massachusetts, United States
Number of Participants With Hematologic Response
Hematologic response defined as: at least 50% improvement in the difference between involved and uninvolved free light chains
Time frame: 6 months
Toxicities
Number of serious adverse events per participant based on Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
Time frame: 100 days
Overall Survival
duration of overall survival measured in days
Time frame: 5 years
Number of Participants With Organ Response
analysis of number of patients with organ response, as defined on page 13 of the detailed protocol for kidney, heart and liver.
Time frame: 5 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells
infusion of previously collected autologous stem cells