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

Primary Purpose

Multiple Myeloma and Plasma Cell Neoplasm

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
filgrastim
melphalan
bone marrow ablation with stem cell support
peripheral blood stem cell transplantation
Sponsored by
Temple University
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Multiple Myeloma and Plasma Cell Neoplasm focused on measuring refractory multiple myeloma, stage I multiple myeloma, stage II multiple myeloma, stage III multiple myeloma, primary systemic amyloidosis

Eligibility Criteria

16 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Fox Chase-Temple Cancer Center CCOP Research Base

Outcomes

Primary Outcome Measures

Overall survival
Time to clinical progression of amyloid symptoms

Secondary Outcome Measures

Full Information

First Posted
November 1, 1999
Last Updated
September 30, 2010
Sponsor
Temple University
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1. Study Identification

Unique Protocol Identification Number
NCT00002810
Brief Title
High-Dose Melphalan Followed by Peripheral Stem Cell Transplant in Treating Patients With Amyloidosis
Official Title
Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2010
Overall Recruitment Status
Completed
Study Start Date
May 1996 (undefined)
Primary Completion Date
May 2006 (Actual)
Study Completion Date
May 2006 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Temple University

4. Oversight

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Multiple Myeloma and Plasma Cell Neoplasm
Keywords
refractory multiple myeloma, stage I multiple myeloma, stage II multiple myeloma, stage III multiple myeloma, primary systemic amyloidosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized

8. Arms, Groups, and Interventions

Intervention Type
Biological
Intervention Name(s)
filgrastim
Intervention Type
Drug
Intervention Name(s)
melphalan
Intervention Type
Procedure
Intervention Name(s)
bone marrow ablation with stem cell support
Intervention Type
Procedure
Intervention Name(s)
peripheral blood stem cell transplantation
Primary Outcome Measure Information:
Title
Overall survival
Title
Time to clinical progression of amyloid symptoms

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kenneth F. Mangan, MD, FACP
Organizational Affiliation
Fox Chase Cancer Center
Official's Role
Study Chair
Facility Information:
Facility Name
Fox Chase-Temple Cancer Center CCOP Research Base
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19111-2442
Country
United States

12. IPD Sharing Statement

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

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