Clinical Trial: Melphalan, Thalidomide, and Dexamethasone in Treating Patients With Newly Diagnosed, Previously Untreated Primary Systemic Amyloidosis
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Risk Adapted Intravenous Melphalan and Adjuvant Thalidomide and Dexamethasone for Untreated Patients With Primary Systemic Amyloidosis
Brief Summary:
RATIONALE: Drugs such as melphalan, thalidomide, and dexamethasone may be effective in treating patients with primary systemic amyloidosis.
PURPOSE: This phase II trial is studying how well giving melphalan together with thalidomide and dexamethasone works in treating patients with primary systemic amyloidosis.
Detailed Summary:
OBJECTIVES:
Primary
- Determine the 2-year and overall progression-free survival of patients with newly diagnosed, previously untreated primary systemic (AL) amyloidosis treated with risk-adapted melphalan followed by thalidomide and dexamethasone.
Secondary
- Determine plasma cell disease response in these patients at 3, 12, and 24 months after treatment with this regimen.
- Determine amyloid-related disease response in these patients at 12 and 24 months after treatment with this regimen.
- Determine the prognostic significance of immunoglobulin light-chain variable-region germline gene expression by AL plasma cell clones in patients treated with this regimen.
- Determine whether there is molecular minimal residual disease at 12 and 24 months in patients achieving a complete hematologic response after treatment with this regimen.
OUTLINE: Patients are stratified according to the extent of amyloid-related disease (low-risk vs high-risk).
- High-risk disease: Patients receive 2 courses of low-dose melphalan IV, dexamethasone, and filgrastim (G-CSF). After 3 months, patients receive thalidomide and dexamethasone if plasma cell disease persists.
- Low-risk disease: Patients receive 1 course of high-dose melphalan IV and G-CSF. Patients then receive thalidomide and dexamethasone as in high-risk disease regimen.
Patients are followed at 3, 12,
Sponsor: Memorial Sloan Kettering Cancer Center
Current Primary Outcome: Overall progression-free survival at 2 years
Original Primary Outcome:
Current Secondary Outcome:
- Plasma cell disease response at 3, 12, and 24 months after treatment
- Amyloid-related disease response at 12 and 24 months after treatment
- Prognostic significance of immunoglobulin light-chain variable-region germline gene expression by AL cell clones
- Molecular minimal residual disease at 12 and 24 months
Original Secondary Outcome:
Information By: Memorial Sloan Kettering Cancer Center
Dates:
Date Received: August 4, 2004
Date Started: May 2002
Date Completion:
Last Updated: January 15, 2013
Last Verified: January 2013