High-Dose Melphalan Followed by Peripheral Stem Cell Transplant in Treating Patients With Amyloidosis

Sponsor

Temple University (Other)

Overall Status

Completed

CT.gov ID

NCT00002810

Collaborator

(none)

Location

120

Duration (Months)

Study Details

Study Description

Brief 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 peripheral 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 giving high-dose melphalan together with peripheral stem cell transplant works in treating patients with primary amyloidosis or amyloidosis associated with multiple myeloma.

Condition or Disease	Intervention/Treatment	Phase
Multiple Myeloma and Plasma Cell Neoplasm	Biological: filgrastim Drug: melphalan Procedure: bone marrow ablation with stem cell support Procedure: peripheral blood stem cell transplantation	Phase 2

Detailed Description

OBJECTIVES:

Assess overall and progression-free survival following high-dose melphalan and autologous peripheral blood stem cell transplantation in patients with primary amyloidosis.
Evaluate the toxic effects associated with this treatment regimen.
Evaluate the function of involved organs, especially the heart, lungs, and nervous system, before and after treatment with this regimen.

OUTLINE: Peripheral blood stem cells (PBSC) are mobilized with granulocyte colony-stimulating factor (G-CSF) for 5 days and then collected by leukapheresis. Patients receive high-dose melphalan on 2 consecutive days, followed by 1 day of rest, then by PBSC transplantation. G-CSF is given from 1 day after transplantation until the neutrophil count is greater than 1,500 for 3 consecutive days.

Patients are followed at 100 days and 1 year post-transplant.

PROJECTED ACCRUAL: A very small number of patients are expected to be accrued over 5-10 years.

Study Design

Study Type:

Interventional

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL)

Study Start Date :

May 1, 1996

Actual Primary Completion Date :

May 1, 2006

Actual Study Completion Date :

May 1, 2006

Outcome Measures

Primary Outcome Measures

Overall survival []
Time to clinical progression of amyloid symptoms []

Eligibility Criteria

Criteria

Ages Eligible for Study:

16 Years to 65 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

DISEASE CHARACTERISTICS:

Primary amyloidosis diagnosed by appropriate amyloid stains or electromicroscopy of abdominal fat, bone marrow, or other target tissues
Pathology reviewed by Temple University
Amyloidosis secondary to any stage of multiple myeloma allowed provided plasma cell concentration in bone marrow is less than 15%
No amyloidosis secondary to rheumatoid arthritis or chronic infection
No familial amyloidosis

PATIENT CHARACTERISTICS:

Age:

16 to 65

Performance status:

Karnofsky 80-100%

Hematopoietic:

Not specified

Hepatic:

Liver function tests less than twice normal
No active liver disease

Renal:

Creatinine clearance greater than 50 mL/min
Nephrotic syndrome allowed

Cardiovascular:

Cardiac evaluation required in patients with left ventricular ejection fraction less than 45% by echocardiogram or MUGA
No poorly controlled hypertension