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Chemotherapy Plus Peripheral Stem Cell Transplant in Treating Patients Who Have Multiple Myeloma or Primary Systemic Amyloidosis

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ClinicalTrials.gov Identifier: NCT00007995
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : February 4, 2013
National Cancer Institute (NCI)
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplant may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells.

PURPOSE: This phase II trial is studying how well chemotherapy and peripheral stem cell transplant work in treating patients with multiple myeloma or primary systemic amyloidosis.

Condition or disease Intervention/treatment Phase
Multiple Myeloma and Plasma Cell Neoplasm Biological: filgrastim Biological: recombinant interferon alfa Biological: sargramostim Drug: busulfan Drug: cyclophosphamide Drug: melphalan Procedure: autologous bone marrow transplantation Procedure: bone marrow ablation with stem cell support Procedure: peripheral blood stem cell transplantation Phase 2

Detailed Description:


  • Determine the response rate in patients with multiple myeloma or primary systemic amyloidosis treated with high-dose chemotherapy with autologous hematopoietic stem cell support.
  • Determine the toxicity of this regimen in these patients.
  • Determine the disease-free survival and overall survival of patients with multiple myeloma treated with this regimen.

OUTLINE: Patients are stratified according to disease response to prior treatment (responsive vs refractory or relapsed) and diagnosis (multiple myeloma vs primary systemic amyloidosis).

Following a course of induction chemotherapy, patients receive filgrastim (G-CSF) subcutaneously (SC) daily until the completion of peripheral blood stem cell (PBSC) harvesting. Patient who do not mobilize sufficient cells undergo bone marrow harvest.

Patients receive melphalan IV over 30 minutes on days -2 and -1. Half of the stored PBSCs and/or bone marrow is reinfused on day 0. Patients receive sargramostim (GM-CSF) daily beginning on day 0 and continuing until blood counts recover. Patients with primary systemic amyloidosis who are not responding to or are unable to tolerate treatment do not proceed to the second course of therapy.

Within 4-6 weeks after receiving melphalan, patients receive oral busulfan on days -8 to -5 followed by cyclophosphamide IV continuously on days -4 and -3. The remaining half of PBSCs and/or bone marrow is reinfused on day 0. Patients receive GM-CSF daily beginning on day 0 and continuing until blood counts recover.

Within 4-12 weeks after receiving the second course of high-dose chemotherapy, multiple myeloma patients receive maintenance therapy consisting of interferon alfa SC 3 days a week, after blood counts recover.

Patients are followed every 3 months for 1 year and then annually for 5 years.

PROJECTED ACCRUAL: Approximately 60-75 patients (25 for responsive disease stratum, 25 for refractory or relapsed disease stratum, and 10-25 for primary systemic amyloidosis stratum) will be accrued for this study within 3 years.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 75 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Study Of High Dose Chemotherapy Followed By Autologous Hematopoietic Stem Cell Support In Patients With Multiple Myeloma And Primary Light Chain Amyloidosis
Study Start Date : July 1999
Actual Primary Completion Date : November 2007
Actual Study Completion Date : May 2008

Primary Outcome Measures :
  1. Disease-free survival at 2 years (patients with responsive disease)

Secondary Outcome Measures :
  1. Duration of hematologic toxicity
  2. Time to an absolute neutrophil count
  3. Platelet independence

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically confirmed multiple myeloma OR
  • Primary systemic amyloidosis resulting in significant organ dysfunction and decreased quality of life
  • Complete or partial response after standard chemotherapy
  • Primary refractory or relapsed multiple myeloma after first-line treatment with standard chemotherapy
  • Ineligible for higher priority national or institutional clinical studies



  • 18 and over

Performance status:

  • ECOG 0-2

Life expectancy:

  • Not specified


  • Not specified


  • Bilirubin less than 2 times normal


  • Creatinine less than 2.5 mg/dL or on stable hemodialysis


  • LVEF at least 45%


  • DLCO at least 60% of predicted OR
  • Approval by pulmonologist


  • HIV negative


Biologic therapy:

  • Concurrent participation in gene therapy trials allowed


  • See Disease Characteristics
  • No other concurrent chemotherapy

Endocrine therapy:

  • No concurrent steroids as antiemetics during chemotherapy
  • No concurrent anticancer hormonal therapy


  • Not specified


  • Not specified


  • No concurrent barbiturates or acetaminophen during chemotherapy
  • Concurrent participation in supportive care trials allowed

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 ClinicalTrials.gov identifier (NCT number): NCT00007995

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United States, New York
Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center
New York, New York, United States, 10032
Sponsors and Collaborators
Herbert Irving Comprehensive Cancer Center
National Cancer Institute (NCI)
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Study Chair: Charles S. Hesdorffer, MD Herbert Irving Comprehensive Cancer Center
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ClinicalTrials.gov Identifier: NCT00007995    
Other Study ID Numbers: CDR0000068361
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: February 4, 2013
Last Verified: July 2007
Keywords provided by National Cancer Institute (NCI):
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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Multiple Myeloma
Neoplasms, Plasma Cell
Immunoglobulin Light-chain Amyloidosis
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
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating