Phase 2 N=10 Treatment

Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis

Multiple Myeloma

Bottom Line

View on ClinicalTrials.gov: NCT00790647 ↗

Enrolled (actual)

Serious AEs

100.0%

Results posted

Feb 2017

Primary outcome: Primary: Number of Participants With Hematologic Response — 6 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: filgrastim (Biological); bortezomib (Drug); melphalan (Drug); Stem Cell Infusion (Procedure)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Boston Medical Center
Primary completion: Dec 2012

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Hematologic Response	6	—
SECONDARY Number of Participants Surviving at 100 Days From Transplant	9	—
SECONDARY Number of Participants Surviving at 1 Year	9	—
SECONDARY Number of Participants Surviving at 2 Years	9	—

Summary

RATIONALE: Giving melphalan and bortezomib before and after a stem cell transplant stops the growth of abnormal cells by stopping them from dividing or killing them. Giving colony-stimulating factors and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy and monoclonal antibody therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy. PURPOSE: This phase II trial is studying how well giving melphalan together with bortezomib followed by stem cell transplant works in treating patients with primary systemic amyloidosis.

Eligibility Criteria

Inclusion criteria

DISEASE CHARACTERISTICS:

Histologically confirmed primary systemic amyloidosis based on the following criteria:
Amyloid light-chain disease
Deposition of amyloid material by congo red stain showing characteristic green birefringence
Monoclonal light chain protein (Bence Jones protein) in the serum or urine, immunohistochemical studies, or serum free light chain assay
Evidence of tissue involvement other than carpal tunnel syndrome (i.e., positive immunohistochemical staining of bone marrow demonstrating clonal plasma cells); tissue amyloid deposits with anti-kappa or anti-lambda anti-serum; evidence for a plasma cell dyscrasia by serum/urine or bone marrow; or overwhelmingly convincing clinical features (e.g., macroglossia) associated with other systemic manifestations

PATIENT CHARACTERISTICS:

Southwest Oncology Group performance status 0-1
Fertile patients must use effective contraception
Left ventricular ejection fraction ≥ 45% by Echocardiogram within the past 60 days
diffusion capacity of lung for carbon monoxide ≥ 50%

PRIOR CONCURRENT THERAPY:

Prior chemotherapy with alkylating agent allowed provided there is no morphological or cytogenetic evidence of myelodysplastic syndromes
Prior total cumulative dose of oral melphalan 30% of bone marrow plasmacytosis, extensive [> 2] lytic lesions, or hypercalcemia)
Not pregnant or nursing
No myocardial infarction within the past 6 months, congestive heart failure, or arrhythmia refractory to therapy
No prior malignancy except for any of the following:
Adequately treated basal cell or squamous cell skin cancer
In situ cervical cancer
Adequately treated stage I or II cancer currently in complete remission
Any cancer from which the patient has been disease-free ≥ 5 years
No advanced (grade 3-4) pre-existing neuropathy
No HIV positivity

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00790647). 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.