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NCT Number: NCT00089167

Melphalan, Thalidomide, and Dexamethasone in Treating Patients With Newly Diagnosed, Previously Untreated Primary Systemic Amyloidosis

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.

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Key information

Conditions

Multiple Myeloma and Plasma Cell Neoplasm Amyloidosis Blood Protein Disorders Cardiovascular Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders Hemostatic Disorders Immune System Diseases Immunoglobulin Light-chain Amyloidosis Immunoproliferative Disorders Lymphoproliferative Disorders Metabolic Diseases Multiple Myeloma Neoplasms Neoplasms by Histologic Type Neoplasms, Plasma Cell Nutritional and Metabolic Diseases Paraproteinemias Proteostasis Deficiencies Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Memorial Sloan-Kettering Cancer Center

New York, 10021, United States

About this study

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, and 24 months.

PROJECTED ACCRUAL: A total of 82 patients will be accrued for this study.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

DISEASE CHARACTERISTICS:

  • Diagnosis of primary systemic (AL) amyloidosis within the past 12 months
  • High- or low-risk disease, determined by the extent of systemic organ involvement with disease and patient age

PATIENT CHARACTERISTICS:

Age

  • 18 and over

Performance status

  • SWOG 0-3

Life expectancy

  • Not specified

Hematopoietic

  • Not specified

Hepatic

  • Not specified

Renal

  • Not specified

Cardiovascular

  • No New York Heart Association class III or IV congestive heart failure
  • No restrictive cardiomyopathy requiring oxygen
  • No myocardial infarction within the past 6 months
  • No symptomatic cardiac arrhythmia within the past 60 days

Other

  • No other active malignancy within the past 5 years except adequately treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, or adequately treated stage I cancer in complete remission

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • Not specified

Chemotherapy

  • No prior chemotherapy for AL amyloidosis

Endocrine therapy

  • Not specified

Radiotherapy

  • Not specified

Surgery

  • Not specified

Other

  • No other prior or concurrent therapy for AL amyloidosis

Treatment and study plan

filgrastim

Biological

Dexamethasone

Drug

melphalan

Drug

thalidomide

Drug

Primary outcomes

  1. Overall progression-free survival at 2 years

Secondary outcomes

  1. Plasma cell disease response at 3, 12, and 24 months after treatment

  2. Amyloid-related disease response at 12 and 24 months after treatment

  3. Prognostic significance of immunoglobulin light-chain variable-region germline gene expression by AL cell clones

  4. Molecular minimal residual disease at 12 and 24 months

Sponsors and collaborators

Lead sponsor

Memorial Sloan Kettering Cancer Center

Other

Collaborators

  • National Cancer Institute (NCI)

Registry information

Official study title

Risk Adapted Intravenous Melphalan and Adjuvant Thalidomide and Dexamethasone for Untreated Patients With Primary Systemic Amyloidosis

Important dates

Study start
2002
Primary completion
2007
First posted
Aug 5, 2004
Registry last updated
Jan 16, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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