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

NCT00458822 | Completed Phase 2 Results

• 2 other identifiers: 07-006MSKCC-07006
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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

Keywords

primary systemic amyloidosis

Outcome Measures

Primary Outcomes (1)

• Hematologic and Organ Response

  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.

  2-3 months post transplant

Study Arms (1)

All Patients

EXPERIMENTAL

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; Drug: dexamethasone

Interventions

bortezomib DRUG

Given IV

All Patients

dexamethasone DRUG

Given orally

All Patients

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

MeSH Terms

Conditions

Multiple Myeloma; Neoplasms, Plasma Cell; Immunoglobulin Light-chain Amyloidosis

Interventions

Bortezomib; Dexamethasone

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Boronic Acids; Acids, Noncarboxylic; Acids; Inorganic Chemicals; Boron Compounds; Organic Chemicals; Pyrazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated

Results Point of Contact

• Title: Dr. Heather Landau
• Organization: Memorial Sloan Kettering Cancer Center

LandauH@mskcc.org

212-639-8808

Study Officials

• Heather Landau, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 9, 2007
• First Posted: April 11, 2007
• Study Start: February 1, 2007
• Primary Completion: March 1, 2015
• Study Completion: March 1, 2015
• Last Updated: August 10, 2016
• Results First Posted: August 10, 2016

Record last verified: 2016-06

Locations

US (1)