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

This study has been completed.
Sponsor:
Information provided by:
Temple University
ClinicalTrials.gov Identifier:
NCT00002810
First received: November 1, 1999
Last updated: September 30, 2010
Last verified: September 2010
  Purpose

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 Intervention 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

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL)

Resource links provided by NLM:


Further study details as provided by Temple University:

Primary Outcome Measures:
  • Overall survival [ Designated as safety issue: No ]
  • Time to clinical progression of amyloid symptoms [ Designated as safety issue: No ]

Study Start Date: May 1996
Study Completion Date: May 2006
Primary Completion Date: May 2006 (Final data collection date for primary outcome measure)
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.

  Eligibility

Ages Eligible for Study:   16 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00002810

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

Additional Information:
No publications provided

Responsible Party: Bone marrow Transplant Program, Temple University Health Systems
ClinicalTrials.gov Identifier: NCT00002810     History of Changes
Other Study ID Numbers: CDR0000064938, TUHSC-2797, NCI-V96-0951
Study First Received: November 1, 1999
Last Updated: September 30, 2010
Health Authority: United States: Federal Government

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:
Amyloidosis
Neoplasms
Multiple Myeloma
Neoplasms, Plasma Cell
Plasmacytoma
Proteostasis Deficiencies
Metabolic Diseases
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Melphalan
Lenograstim
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antineoplastic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on April 23, 2014