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Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain 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: NCT00017680
Recruitment Status : Completed
First Posted : June 6, 2001
Last Update Posted : June 9, 2008
Information provided by:
Office of Rare Diseases (ORD)

Brief Summary:

OBJECTIVES: I. Determine the response, disease-free survival, and overall survival of patients with primary light chain amyloidosis treated with high-dose melphalan and autologous stem cell transplantation.

II. Determine the toxicity of this regimen in these patients.

Condition or disease Intervention/treatment Phase
Amyloidosis Drug: Melphalan Procedure: Autologous Stem Cell Transplantation Phase 2

Detailed Description:

PROTOCOL OUTLINE: Patients may receive induction chemotherapy before study entry. Patients then receive filgrastim (G-CSF) or another growth factor for 4-6 days as peripheral blood stem cell (PBSC) mobilization. PBSC (or bone marrow) is harvested over 2-3 days.

Patients receive high-dose melphalan IV over 30 minutes twice daily on days -2 and -1. PBSC and/or bone marrow is reinfused on day 0. Patients receive G-CSF beginning on day 0 and continuing until blood counts recover. This course may be repeated 4-12 weeks later.

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

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Study Type : Interventional  (Clinical Trial)
Enrollment : 25 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in
Study Start Date : July 1999
Actual Primary Completion Date : April 2004
Actual Study Completion Date : April 2004

Intervention Details:
  • Drug: Melphalan
    100 mg/m2 twice a day from Days -2 and -1 as induction therapy
  • Procedure: Autologous Stem Cell Transplantation
    Bone marrow and peripheral blood stem cells harvested

Primary Outcome Measures :
  1. Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction

Secondary Outcome Measures :
  1. Toxicity of high dose chemotherapy regimen

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


Disease Characteristics

  • Histologically confirmed primary amyloidosis
  • Ineligible for other high priority national or international study

Prior/Concurrent Therapy

  • Biologic therapy: Concurrent participation in gene therapy trials allowed
  • Chemotherapy: Prior chemotherapy allowed No other concurrent chemotherapy
  • Endocrine therapy: No concurrent steroids unless given with amphotericin B, for adrenal failure, or for septic shock No concurrent hormones except for non-disease-related conditions (e.g., insulin for diabetes)
  • Other: No concurrent barbiturates or acetaminophen Concurrent participation in supportive care trials allowed

Patient Characteristics

  • Performance status: ECOG 0-3
  • Hepatic: Bilirubin less than 2 times normal
  • Renal: Creatinine less than 2.5 mg/dL OR On stable hemodialysis
  • Pulmonary: DLCO at least 60% predicted OR Clearance by pulmonologist
  • Other: HIV negative

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): NCT00017680

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United States, New York
Herbert Irving Comprehensive Cancer Center
New York, New York, United States, 10032
Sponsors and Collaborators
Herbert Irving Comprehensive Cancer Center
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Study Chair: Charles S. Hesdorffer Herbert Irving Comprehensive Cancer Center

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Responsible Party: Charles Hesdorffer, MD, Columbia University Identifier: NCT00017680     History of Changes
Other Study ID Numbers: 199/15927
First Posted: June 6, 2001    Key Record Dates
Last Update Posted: June 9, 2008
Last Verified: June 2008
Keywords provided by Office of Rare Diseases (ORD):
arthritis & connective tissue diseases
genetic diseases and dysmorphic syndromes
hematopoietic/lymphoid cancer
oncologic disorders
plasma cell neoplasm
primary systemic amyloidosis
rare disease
Additional relevant MeSH terms:
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Proteostasis Deficiencies
Metabolic Diseases
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs