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