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

This study has been completed.
Sponsor:
Collaborator:
Information provided by:
Memorial Sloan-Kettering Cancer Center
ClinicalTrials.gov Identifier:
NCT00089167
First received: August 4, 2004
Last updated: January 15, 2013
Last verified: January 2013
  Purpose

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.


Condition Intervention Phase
Multiple Myeloma and Plasma Cell Neoplasm
Biological: filgrastim
Drug: dexamethasone
Drug: melphalan
Drug: thalidomide
Phase 2

Study Type: Interventional
Study Design: Masking: Open Label
Primary Purpose: Treatment
Official Title: Risk Adapted Intravenous Melphalan and Adjuvant Thalidomide and Dexamethasone for Untreated Patients With Primary Systemic Amyloidosis

Resource links provided by NLM:


Further study details as provided by Memorial Sloan-Kettering Cancer Center:

Primary Outcome Measures:
  • Overall progression-free survival at 2 years [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Plasma cell disease response at 3, 12, and 24 months after treatment [ Designated as safety issue: No ]
  • Amyloid-related disease response at 12 and 24 months after treatment [ Designated as safety issue: No ]
  • Prognostic significance of immunoglobulin light-chain variable-region germline gene expression by AL cell clones [ Designated as safety issue: No ]
  • Molecular minimal residual disease at 12 and 24 months [ Designated as safety issue: No ]

Study Start Date: May 2002
Primary Completion Date: December 2007 (Final data collection date for primary outcome measure)
Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00089167

Locations
United States, New York
Memorial Sloan-Kettering Cancer Center
New York, New York, United States, 10021
Sponsors and Collaborators
Memorial Sloan-Kettering Cancer Center
Investigators
Principal Investigator: Raymond L. Comenzo, MD Memorial Sloan-Kettering Cancer Center
Principal Investigator: Madhav Dhodapkar, MD Memorial Sloan-Kettering Cancer Center
  More Information

Additional Information:
Publications:
Cohen AD, Zhou P, Reich L, et al.: Risk-adapted intravenous melphalan followed by adjuvant dexamethasone (D) and thalidomide (T) for newly diagnosed patients with Systemic AL Amyloidosis (AL): interim results of a phase II study. [Abstract] Blood 104 (11): A-542, 2004.

ClinicalTrials.gov Identifier: NCT00089167     History of Changes
Other Study ID Numbers: 02-031, MSKCC-02031
Study First Received: August 4, 2004
Last Updated: January 15, 2013
Health Authority: United States: Federal Government

Keywords provided by Memorial Sloan-Kettering Cancer Center:
primary systemic amyloidosis

Additional relevant MeSH terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Amyloidosis
Plasmacytoma
Neoplasms by Histologic Type
Neoplasms
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Proteostasis Deficiencies
Metabolic Diseases
Dexamethasone acetate
Dexamethasone
Dexamethasone 21-phosphate
Thalidomide
Melphalan
BB 1101
Anti-Inflammatory Agents
Therapeutic Uses
Pharmacologic Actions
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents

ClinicalTrials.gov processed this record on September 29, 2014