Clinical Trial: Bortezomib and Dexamethasone Followed by High-Dose Melphalan and Stem Cell Transplantation for Primary (AL) Amyloidosis

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Phase II Trial of Induction Therapy With Bortezomib and Dexamethasone Followed by High-Dose Melphalan and Stem Cell Transplantation in Patients With AL Amyloidosis

Brief Summary:

The drugs dexamethasone and bortezomib are both FDA-approved for the treatment of multiple myeloma, a disease very similar to amyloidosis. However, they are currently investigational for the treatment of amyloidosis.

We want to find out if the addition of dexamethasone and bortezomib to standard high dose chemotherapy and stem cell transplant can help improve response.

Standard treatment includes four steps: 1) Stem Cell Mobilization (standard) 2) Stem Cell Collection (standard) 3) Conditioning Regimen (Melphalan chemotherapy). The conditioning regimen helps to kill the abnormal cells in the body and makes room in the bone marrow for new blood stem cells to grow. 4) Stem Cell Infusion

Participants in this study will have an additional treatment step called "induction therapy", designed as the first step towards reducing the number of abnormal cells in the body. Two cycles of the investigational drugs bortezomib and dexamethasone will be given during induction therapy. In addition, bortezomib will given as part of the conditioning regimen, in addition to the standard melphalan chemotherapy.


Detailed Summary:

The drugs dexamethasone and bortezomib are both FDA-approved drugs for the treatment of multiple myeloma, a disease very similar to amyloidosis. However, they are currently investigational for the treatment of amyloidosis.

The investigators want to find out if the addition of dexamethasone and bortezomib to standard treatment of high dose chemotherapy and stem cell transplant can help improve response to treatment.


Sponsor: Boston Medical Center

Current Primary Outcome:

  • Number of Participants With Disease Response [ Time Frame: One year ]
    Complete response: Normal serum free light chain ratio and Negative serum and urine immunofixation electrophoresis Very good partial response: Difference in serum free light chains less than 40 mg/L Partial Response: >50% Reduction in the difference in serum free light chains
  • Number of Participants Surviving at 100 Days Post Transplant [ Time Frame: 100 days ]
  • Number of Participants Proceeding to Transplant Following Induction [ Time Frame: 2 months ]


Original Primary Outcome: Disease Response [ Time Frame: One year ]

Current Secondary Outcome: Number of Participants Surviving at 5 Years [ Time Frame: 5 years ]

Original Secondary Outcome:

  • To determine toxicities [ Time Frame: 100 Days from transplant date ]
  • To determine overall survival [ Time Frame: 5 years ]


Information By: Boston Medical Center

Dates:
Date Received: November 2, 2009
Date Started: September 2009
Date Completion: December 2040
Last Updated: April 6, 2017
Last Verified: April 2017