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Completed

NCT Number: NCT00458822

Melphalan and Autologous Stem Cell Transplant Followed By Bortezomib and Dexamethasone in Treating Patients With Previously Untreated Systemic Amyloidosis

The purpose of this study in patients needing treatment for AL amyloidosis is to see how well treatment with IV melphalan works and then, if some clonal plasma cells are still present about 2 to 3 months after melphalan treatment, to see how well treatment with bortezomib and dexamethasone works to reduce the rest of the clonal plasma cell disease.

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Key information

Who can participate

Healthy volunteers accepted: No

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

DISEASE CHARACTERISTICS:

  • Histologically confirmed amyloidosis
  • Diagnosed within the past 12 months
  • Clonal plasma cell disorder, as demonstrated by any of the following:
  • Presence of M-protein in serum and/or urine by immunofixation and/or serum free light chain assay
  • Clonal population of plasma cells in the bone marrow based on kappa/lambda staining of a marrow biopsy
  • Negative genetic testing for hereditary forms of amyloidosis
  • No amyloid-specific syndrome (e.g., carpal tunnel syndrome or skin purpura) as the only evidence of disease
  • Vascular amyloidosis only in a bone marrow biopsy specimen or in plasmacytoma is not indicative of systemic amyloidosis
  • No advanced cardiac amyloidosis
  • Must have symptomatic involvement of no more than 2 of the following visceral organ systems:
  • Kidneys
  • Liver/gastrointestinal
  • Peripheral/autonomic nervous system
  • Heart
  • No persistent pleural effusions
  • No clinically overt multiple myeloma with > 30% plasma cells in the bone marrow or lytic bone lesions
  • Able to undergo autologous stem cell transplantation

PATIENT CHARACTERISTICS:

  • SWOG performance status 0-3
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • Bilirubin < 2.0 mg/dL
  • Creatinine clearance < 51 mL/min allowed
  • LVEF > 45% by echocardiogram
  • No New York Heart Association class III-IV congestive heart failure
  • No history of cardiac syncope
  • No recurrent symptomatic arrhythmias
  • No oxygen-dependent restrictive cardiomyopathy
  • No myocardial infarction within the past 6 months
  • Pulmonary diffusion capacity > 50% predicted by pulmonary function testing
  • No uncontrolled infection
  • No other active malignancy, except for any of the following:
  • Adequately treated basal cell or squamous cell skin cancer
  • In situ cervical cancer
  • Adequately treated stage I cancer from which the patient is currently in complete remission
  • Any other cancer from which the patient has been disease-free for 5 years
  • No hypersensitivity to bortezomib, boron, or mannitol
  • No HIV positivity
  • No serious medical or psychiatric illness that would preclude study compliance

PRIOR CONCURRENT THERAPY:

  • At least 14 days since prior investigational drugs
  • No prior therapy for monoclonal plasma disease

Treatment and study plan

bortezomib

Drug

Given IV

Dexamethasone

Drug

Given orally

Primary outcomes

  1. Hematologic and Organ Response

    Time frame: 2-3 months post transplant

    patients will be assessed for hematologic response (the response of the clonal plasma cell disease). If the plasma cell disease persists, then they will receive 6 cycles of adjuvant therapy with bortezomib and dexamethasone; patients with peripheral neuropathy will receive dexamethasone alone because of the risk of neuropathy associated with bortezomib. Symptomatic organ involvement with amyloid as defined below. Patients must have symptomatic involvement of no more than 2 of the following 4 visceral organ-systems: kidneys, liver/GI, peripheral/autonomic nervous system, and heart.

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 With Stem Cell Transplant and Adjuvant Bortezomib and Dexamethasone for Recently Diagnosed Untreated Patients With Systemic Light-Chain (AL) Amyloidosis

Important dates

Study start
2007
Primary completion
2015
Study completion
2015
First posted
Apr 11, 2007
Registry last updated
Aug 10, 2016

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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