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

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ClinicalTrials.gov Identifier: NCT00458822
Recruitment Status : Completed
First Posted : April 11, 2007
Results First Posted : August 10, 2016
Last Update Posted : August 10, 2016
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Memorial Sloan Kettering Cancer Center

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Multiple Myeloma and Plasma Cell Neoplasm Drug: bortezomib Drug: dexamethasone Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Study Start Date : February 2007
Actual Primary Completion Date : March 2015
Actual Study Completion Date : March 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: All Patients
All patients treated with Melphalan with Stem Cell Transplant and Adjuvant Bortezomib and Dexamethasone for Recently Diagnosed Untreated Patients with Systemic Light-Chain (AL) Amyloidosis
 Drug: bortezomib
Given IV

Drug: dexamethasone
Given orally


Outcome Measures
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Primary Outcome Measures :
  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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00458822


Locations
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United States, New York
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Sponsors and Collaborators
Memorial Sloan Kettering Cancer Center
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Heather Landau, MD Memorial Sloan Kettering Cancer Center
More Information
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Responsible Party: Memorial Sloan Kettering Cancer Center
ClinicalTrials.gov Identifier: NCT00458822    
Other Study ID Numbers: 07-006
MSKCC-07006
First Posted: April 11, 2007    Key Record Dates
Results First Posted: August 10, 2016
Last Update Posted: August 10, 2016
Last Verified: June 2016
Keywords provided by Memorial Sloan Kettering Cancer Center:
primary systemic amyloidosis
Additional relevant MeSH terms:
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Multiple Myeloma
Neoplasms, Plasma Cell
Plasmacytoma
Amyloidosis
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
Bortezomib
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal