Back to resultsStudy of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain Amyloidosis
Primary Purpose
Amyloidosis
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Melphalan
Autologous Stem Cell Transplantation
Sponsored by
About this trial
This is an interventional treatment trial for Amyloidosis focused on measuring amyloidosis, arthritis & connective tissue diseases, genetic diseases and dysmorphic syndromes, hematopoietic/lymphoid cancer, oncologic disorders, plasma cell neoplasm, primary systemic amyloidosis, rare disease
Eligibility Criteria
PROTOCOL ENTRY CRITERIA: 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
Sites / Locations
- Herbert Irving Comprehensive Cancer Center
Outcomes
Primary Outcome Measures
Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction
Secondary Outcome Measures
Toxicity of high dose chemotherapy regimen
Full Information
NCT ID
NCT00017680
First Posted
June 6, 2001
Last Updated
June 6, 2008
Sponsor
Herbert Irving Comprehensive Cancer Center
1. Study Identification
Unique Protocol Identification Number
NCT00017680
Brief Title
Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain Amyloidosis
Official Title
Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in
Study Type
Interventional
2. Study Status
Record Verification Date
June 2008
Overall Recruitment Status
Completed
Study Start Date
July 1999 (undefined)
Primary Completion Date
April 2004 (Actual)
Study Completion Date
April 2004 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Herbert Irving Comprehensive Cancer Center
4. Oversight
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyloidosis
Keywords
amyloidosis, arthritis & connective tissue diseases, genetic diseases and dysmorphic syndromes, hematopoietic/lymphoid cancer, oncologic disorders, plasma cell neoplasm, primary systemic amyloidosis, rare disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
25 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Melphalan
Intervention Description
100 mg/m2 twice a day from Days -2 and -1 as induction therapy
Intervention Type
Procedure
Intervention Name(s)
Autologous Stem Cell Transplantation
Intervention Description
Bone marrow and peripheral blood stem cells harvested
Primary Outcome Measure Information:
Title
Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction
Secondary Outcome Measure Information:
Title
Toxicity of high dose chemotherapy regimen
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
PROTOCOL ENTRY CRITERIA:
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Charles S. Hesdorffer
Organizational Affiliation
Herbert Irving Comprehensive Cancer Center
Official's Role
Study Chair
Facility Information:
Facility Name
Herbert Irving Comprehensive Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain Amyloidosis
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