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

This study has been completed.
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Memorial Sloan Kettering Cancer Center
ClinicalTrials.gov Identifier:
NCT00458822
First received: April 9, 2007
Last updated: June 29, 2016
Last verified: June 2016
  Purpose
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 Intervention Phase
Multiple Myeloma and Plasma Cell Neoplasm
Drug: bortezomib
Drug: dexamethasone
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


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

Primary Outcome Measures:
  • Hematologic and Organ Response [ Time Frame: 2-3 months post transplant ] [ Designated as safety issue: No ]
    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.


Enrollment: 40
Study Start Date: February 2007
Study Completion Date: March 2015
Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years   (Adult, Senior)
Genders Eligible for Study:   Both
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
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: NCT00458822

Locations
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
Principal Investigator: Heather Landau, MD Memorial Sloan Kettering Cancer Center
  More Information

Responsible Party: Memorial Sloan Kettering Cancer Center
ClinicalTrials.gov Identifier: NCT00458822     History of Changes
Other Study ID Numbers: 07-006  MSKCC-07006 
Study First Received: April 9, 2007
Results First Received: January 4, 2016
Last Updated: June 29, 2016
Health Authority: United States: Institutional Review Board

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

Additional relevant MeSH terms:
Multiple Myeloma
Amyloidosis
Neoplasms, Plasma Cell
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
Melphalan
Bortezomib
BB 1101
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents

ClinicalTrials.gov processed this record on December 09, 2016