Phase 2 N=40 Treatment

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

Multiple Myeloma and Plasma Cell Neoplasm

Bottom Line

View on ClinicalTrials.gov: NCT00458822 ↗

Enrolled (actual)

Serious AEs

50.0%

Results posted

Aug 2016

Primary outcome: Primary: Hematologic and Organ Response — 11; 7; 18 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: bortezomib (Drug); dexamethasone (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Memorial Sloan Kettering Cancer Center
Primary completion: Mar 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Hematologic and Organ Response	11; 7; 18	—

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.

Eligibility 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 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00458822). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.