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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00458822
• Date of registration: 09/04/2007
• Prospective Registration: No
• Primary sponsor: Memorial Sloan Kettering Cancer Center
• Public title: Melphalan and Autologous Stem Cell Transplant Followed By Bortezomib and Dexamethasone in Treating Patients With Previously Untreated Systemic Amyloidosis
• Scientific 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
• Date of first enrolment: February 2007
• Target sample size: 40
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00458822
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Heather Landau, MD
• Affiliation: Memorial Sloan Kettering Cancer Center

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Multiple Myeloma and Plasma Cell Neoplasm

Intervention(s)

Drug: bortezomib

Drug: dexamethasone

Primary Outcome(s)

Hematologic and Organ Response [Time Frame: 2-3 months post transplant]

Secondary ID(s)

07-006

MSKCC-07006

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Cancer Institute (NCI)

Results

• Results available: Yes
• Date Posted: 10/08/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00458822