NCT00520767

Bortezomib, Melphalan, and Dexamethasone in Treating Patients With Primary Amyloidosis or Light Chain Deposition Disease

A Multicenter Phase II Trial of Bortezomib (Velcade), Melphalan, and Dexamethasone (V-MD) in Patients With Symptomatic AL-Amyloidosis or Light Chain Deposition Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 35 (actual)
• Sponsor: Barbara Ann Karmanos Cancer Institute · Academic / Other
• Sex: All
• Age: 18 Years – 120 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Primary Systemic Amyloidosis
• Light Chain Deposition Disease

Interventions

Type	Name	Description
DRUG	bortezomib	Bortezomib 1.3 mg/m2 days 1, 8, 15, 22
DRUG	dexamethasone	Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23
DRUG	melphalan	Melphalan 9 mg/m2/day days 1-4
GENETIC	microarray analysis	≤28 days prior to enrollment
OTHER	flow cytometry	Day 1 of cycles 6, 12, 18 and at end of study.
OTHER	laboratory biomarker analysis	≤28 days prior to enrollment
PROCEDURE	quality-of-life assessment	Start of each cycle

Timeline

• Start date: 2007-09-01
• Primary completion: 2010-10-01
• Completion: 2019-06-06
• First posted: 2007-08-27
• Last updated: 2023-12-22
• Results posted: 2015-03-30

Locations

7 sites across 1 country: United States