Bortezomib, Melphalan, and Dexamethasone in Treating Patients With Primary Amyloidosis or Light Chain Deposition Disease
Condition: Primary Systemic Amyloidosis; Light Chain Deposition Disease
MeSH terms: Immunoglobulin Light-chain Amyloidosis; Multiple Myeloma; Amyloidosis
Study type: Interventional
Phase: Phase 2

Summary: 
      RATIONALE: Giving bortezomib together with melphalan and dexamethasone may be an effective
      treatment for primary amyloidosis and light chain deposition disease.

      PURPOSE: This phase II trial is studying how well giving bortezomib together with melphalan
      and dexamethasone works in treating patients with primary amyloidosis or light chain
      deposition disease.
    

Detailed description: 
      OBJECTIVES:

      Primary

        -  Determine the complete hematologic response rate at 12 months.

      Secondary

        -  Determine the overall hematologic response rate.

        -  Determine the organ response rate.

        -  Determine time to treatment failure.

        -  Determine the overall survival.

      OUTLINE: This is a multicenter study.

      Patients receive oral melphalan on days 1-4, bortezomib IV on days 1, 8, 15, and 22, and
      dexamethasone orally or IV on days 1, 2, 8, 9, 15, 16, 22, and 23. Treatment repeats every
      4-6 weeks for up to 20 courses in the absence of disease progression or unacceptable
      toxicity.

      Blood, urine, and bone marrow aspirates are collected at baseline and periodically after
      treatment to permit the correlation of clinical results with measured molecular events. A
      single baseline peripheral blood DNA sample is collected for future association studies
      linking disease onset, progression, and response to administered therapy with single
      nucleotide polymorphisms. Blood plasma and urine samples are evaluated for proteomic markers
      associated with disease progression and therapeutic response. Peripheral blood RNA samples
      are evaluated for transcriptional response to treatment of peripheral blood lymphocytes. Bone
      marrow aspirates are collected to extract plasma cells by flow cytometry for gene expression
      profiling.

      Quality of life is assessed at the beginning of each course.
    

Intervention name(s): bortezomib; dexamethasone; melphalan; microarray analysis; flow cytometry; laboratory biomarker analysis; quality-of-life assessment
Intervention type(s): Drug; Drug; Drug; Genetic; Other; Other; Procedure
Intervention description(s): Bortezomib 1.3 mg/m2 days 1, 8, 15, 22; Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23; Melphalan 9 mg/m2/day days 1-4; ≤28 days prior to enrollment; Day 1 of cycles 6, 12, 18 and at end of study.; ≤28 days prior to enrollment; Start of each cycle
Other intervention name(s): Velcade; Dexasone; Decadron; Diodex; Hexadrol; Maxidex; Dexamethasone Sodium Phosphate; Dexamethasone Acetate; Alkeran®; L-PAM; L-Sarcolysin; Phenylalanine Mustard
Intervention MeSH terms: Dexamethasone; Dexamethasone acetate; Bortezomib; Melphalan; Dexamethasone 21-phosphate; BB 1101
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: primary systemic amyloidosis; light chain deposition disease