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

NCT ID: NCT00458822

Last Updated: 2016-08-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-02-28
• Study Completion Date: 2015-03-31

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.

Conditions

Multiple Myeloma and Plasma Cell Neoplasm

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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

Group Type: EXPERIMENTAL

bortezomib

Intervention Type: DRUG

Given IV

dexamethasone

Intervention Type: DRUG

Given orally

Interventions

bortezomib

Given IV

Intervention Type: DRUG

dexamethasone

Given orally

Intervention Type: DRUG

Eligibility Criteria

Inclusion 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

Memorial Sloan Kettering Cancer Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Heather Landau, MD

Role: PRINCIPAL_INVESTIGATOR

Memorial Sloan Kettering Cancer Center

Locations

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Countries

United States

Other Identifiers

MSKCC-07006

Identifier Type: -

Identifier Source: secondary_id

07-006

Identifier Type: -

Identifier Source: org_study_id