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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00002810
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : October 1, 2010
Information provided by:
Temple University

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:


  • 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.

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Study Type : Interventional  (Clinical Trial)
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 1996
Actual Primary Completion Date : May 2006
Actual Study Completion Date : May 2006

Primary Outcome Measures :
  1. Overall survival
  2. Time to clinical progression of amyloid symptoms

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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



  • 16 to 65

Performance status:

  • Karnofsky 80-100%


  • Not specified


  • Liver function tests less than twice normal
  • No active liver disease


  • Creatinine clearance greater than 50 mL/min
  • Nephrotic syndrome allowed


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


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


  • 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


Biologic therapy:

  • Not specified


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

Endocrine therapy:

  • Corticosteroids discontinued at least 6 weeks prior to transplantation


  • No prior radiotherapy


  • Not specified

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00002810

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United States, Pennsylvania
Fox Chase-Temple Cancer Center CCOP Research Base
Philadelphia, Pennsylvania, United States, 19111-2442
Sponsors and Collaborators
Temple University
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Study Chair: Kenneth F. Mangan, MD, FACP Fox Chase Cancer Center

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Responsible Party: Bone marrow Transplant Program, Temple University Health Systems Identifier: NCT00002810     History of Changes
Other Study ID Numbers: CDR0000064938
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: October 1, 2010
Last Verified: September 2010
Keywords provided by Temple University:
refractory multiple myeloma
stage I multiple myeloma
stage II multiple myeloma
stage III multiple myeloma
primary systemic amyloidosis
Additional relevant MeSH terms:
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Immunosuppressive Agents
Multiple Myeloma
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Proteostasis Deficiencies
Metabolic Diseases
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Immunologic Factors
Physiological Effects of Drugs