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

September 30, 2010 updated by: Temple University

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19111-2442
        • Fox Chase-Temple Cancer Center CCOP Research Base

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Pulmonary:

  • FEV_1 and DLCO greater than 50% of predicted, or pulmonary evaluation required
  • No chronic obstructive pulmonary disease

Other:

  • No history of serious coagulopathy, hemorrhage, or bleeding
  • No active infection
  • No other serious comorbid disease (e.g., poorly controlled diabetes)
  • No pregnant women
  • Adequate contraception required of fertile women

PRIOR CONCURRENT THERAPY:

Biologic therapy:

  • Not specified

Chemotherapy:

  • More than 12 monthly cycles of prior alkylating agent chemotherapy discouraged

Endocrine therapy:

  • Corticosteroids discontinued at least 6 weeks prior to transplantation

Radiotherapy:

  • No prior radiotherapy

Surgery:

  • Not specified

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Overall survival
Time to clinical progression of amyloid symptoms

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Temple University

Investigators

  • Study Chair: Kenneth F. Mangan, MD, FACP, Fox Chase Cancer Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 1996

Primary Completion (Actual)

May 1, 2006

Study Completion (Actual)

May 1, 2006

Study Registration Dates

First Submitted

November 1, 1999

First Submitted That Met QC Criteria

January 26, 2003

First Posted (Estimate)

January 27, 2003

Study Record Updates

Last Update Posted (Estimate)

October 1, 2010

Last Update Submitted That Met QC Criteria

September 30, 2010

Last Verified

September 1, 2010

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CDR0000064938
  • TUHSC-2797
  • NCI-V96-0951

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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