Basic Trial Information
Trial Description
     Summary
     Further Trial Information
     Eligibility Criteria
Trial Contact Information

Basic Trial Information

Trial Description

Summary

RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more plasma cells are killed. By reducing the number of plasma cells, the disease may progress more slowly.

PURPOSE: This phase II trial is studying how well autologous stem cell transplant works in treating patients with persistent or recurrent primary systemic (AL) amyloidosis.

Further Study Information

OBJECTIVES:

• Determine the feasibility and tolerability of second autologous stem cell transplantation in patients with persistent or recurrent AL amyloidosis.

• Determine the response rate and durability of response in patients treated with this regimen.

• Determine immune reconstitution in patients treated with this regimen.

OUTLINE:

• Mobilization: Patients receive filgrastim (G-CSF) subcutaneously (SC) once daily beginning before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection.

• Preparative regimen: Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2.

• Autologous stem cell transplantation: Autologous stem cells are reinfused on day 0.

Patients are followed at 6 months, 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 19 patients will be accrued for this study within 5-6 years.

Eligibility Criteria

DISEASE CHARACTERISTICS:

• Histologically confirmed AL amyloidosis

• Persistent or recurrent disease after 1 course of prior high-dose chemotherapy

• Previously treated with autologous stem cell transplantation

• Significant initial improvement in organ function after prior high-dose melphalan, defined by at least 1 of the following:

• Complete hematologic remission (e.g., absence of monoclonal spike by immunofixation in serum and urine AND less then 5% plasma cells in bone marrow with no clonal predominance) OR partial hematologic response (e.g., any decrease in serum or urine monoclonal protein OR decrease in bone marrow plasmacytosis)

• Greater than 50% reduction in proteinuria with preservation of creatinine clearance

• Greater than 50% reduction in alkaline phosphatase OR at least 2 cm decrease in liver size by physical exam

• Subjective neurologic improvement, as confirmed by neurologist

• Cardiac stabilization of disease confirmed by echocardiography defined as less than 2 mm increase in mean wall thickness and/or less than 20 g increase in left ventricular mass

• Improvement in performance status* NOTE: *This criteria alone does not constitute significant improvement in organ function

• No myelodysplastic syndromes

• No abnormal bone marrow cytogenetics

• Prior stem cell yield must have been ≥ 2 x 10^6 CD34+ cells/kg

PATIENT CHARACTERISTICS:

Age

• 18 to 65

Performance status

• SWOG 0-2

Life expectancy

• More than 6 months

Hematopoietic

• See Disease Characteristics

Hepatic

• See Disease Characteristics

Renal

• See Disease Characteristics

Cardiovascular

• See Disease Characteristics

• LVEF ≥ 45% by MUGA or echocardiogram

Pulmonary

• DLCO ≥ 50%

Other

• Not pregnant or nursing

• Fertile patients must use effective contraception

• Acceptable toxicity from first transplantation, confirmed by the transplant team

• HIV negative

• No other concurrent malignancy except treated skin cancer

PRIOR CONCURRENT THERAPY:

Biologic therapy

• See Disease Characteristics

Chemotherapy

• See Disease Characteristics

• No chemotherapy after first transplantation

Endocrine therapy

• Not specified

Radiotherapy

• Not specified

Surgery

• Not specified

Trial Contact Information

Trial Lead Organizations/Sponsors

Boston University Cancer Research Center

Karen Quillen

Principal Investigator

Link to the current ClinicalTrials.gov record.
NLM Identifer NCT00075608
Information obtained from ClinicalTrials.gov on December 14, 2011

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