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High-Dose Melphalan Plus Peripheral Stem Cell Transplantation in Treating Patients With Primary Systemic Amyloidosis
This study has been completed.
First Received: November 1, 1999   Last Updated: February 6, 2009   History of Changes
Sponsors and Collaborators: Eastern Cooperative Oncology Group
National Cancer Institute (NCI)
Information provided by: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00003353
  Purpose

RATIONALE: High-dose chemotherapy may destroy the amyloid-producing cells in bone marrow. Peripheral stem cell transplantation

PURPOSE: Phase II trial to study the effectiveness of high dose melphalan plus peripheral stem cell transplantation in treating patients who have primary systemic amyloidosis.


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 II

Study Type: Interventional
Study Design: Treatment
Official Title: A Phase II Study of High-Dose Melphalan With Hematopoietic Stem Cell Reconstitution for Patients With Primary Systemic Amyloidosis

Resource links provided by NLM:


Further study details as provided by National Cancer Institute (NCI):

Study Start Date: July 1998
Detailed Description:

OBJECTIVES: I. Assess the response rate and overall survival of patients with biopsy proven primary amyloidosis following treatment with myeloablative chemotherapy and hematopoietic stem cell reconstitution. II. Evaluate the toxicity of high dose melphalan in this patient population.

OUTLINE: Patients receive filgrastim (G-CSF) subcutaneously daily beginning on day 1 of the peripheral blood stem cell (PBSC) collection period and continuing until PBSC collection is completed. PBSC are collected beginning on day 5 of the collection period and continuing until the final target cell count is reached or for up to a maximum of 7 collections. If sufficient PBSCs are not harvested within a maximum of 7 collections, the patient is removed from the study. Within 30 days of PBSC collection, patients receive melphalan IV on day -1 of the infusion period and PBSC infusion on day 0. The infusion period continues until day 30. Patients receive G-CSF subcutaneously daily starting on day 1 and continuing until blood counts recover. Patients are followed every 3 months for 2 years, every 3 months for 3 additional years, and yearly thereafter.

PROJECTED ACCRUAL: A maximum of 33 patients will be accrued for this study over 2 years.

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

DISEASE CHARACTERISTICS: Histologically confirmed primary amyloidosis Must have presence of paraprotein in serum or urine determined by immunoelectrophoresis/immunofixation No primary amyloidosis manifested only by carpal tunnel syndrome or purpura No history of secondary, familial, or localized amyloidosis No evidence of overt multiple myeloma: Lytic bone disease or pathological fractures OR At least 30% plasma cells in bone marrow

PATIENT CHARACTERISTICS: Age: 18 to 70 Performance status: ECOG 0-2 Life expectancy: Not specified

Hematopoietic: Absolute granulocyte count at least 1000/mm3 Platelet count at least 100,000/mm3 Hepatic:

Bilirubin no greater than 2.0 mg/dL Alkaline phosphatase no greater than 1000 u/L or less than 4 times upper limit of normal Renal: Creatinine no greater than 2.0 mg/dL Cardiovascular: Confirmed by echocardiogram: Ejection fraction at least 50% Interventricular septal thickness no greater than 15 mm No New York Heart Association classification II-IV Pulmonary: DLCO at least 50% FVC at least 60% FEV1 at least 55% Other: Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception No active infection No other malignancy within the past 5 years except surgically treated carcinoma in situ of the cervix, nonmelanoma skin cancer, or indolent prostate cancer No known sensitivity to E. coli derivatives

PRIOR CONCURRENT THERAPY: Biologic therapy: Prior interferon allowed Chemotherapy: At least 4 weeks since prior melphalan Lifetime cumulative dose of melphalan no greater than 150 mg No greater than 2 prior courses of chemotherapy Endocrine therapy: Prior dexamethasone allowed Radiotherapy: Not specified Surgery: Not specified

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00003353

Locations
United States, Arizona
CCOP - Scottsdale Oncology Program
Scottsdale, Arizona, United States, 85259-5404
United States, Florida
H. Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, United States, 33612
Sylvester Cancer Center, University of Miami
Miami, Florida, United States, 33136
United States, Illinois
CCOP - Evanston
Evanston, Illinois, United States, 60201
CCOP - Illinois Oncology Research Association
Peoria, Illinois, United States, 61602
United States, Indiana
Indiana University Cancer Center
Indianapolis, Indiana, United States, 46202-5265
Veterans Affairs Medical Center - Indianapolis (Roudebush)
Indianapolis, Indiana, United States, 46202
United States, Massachusetts
New England Medical Center Hospital
Boston, Massachusetts, United States, 02111
United States, Minnesota
CCOP - Metro-Minnesota
Saint Louis Park, Minnesota, United States, 55416
Mayo Clinic Cancer Center
Rochester, Minnesota, United States, 55905
United States, New York
University of Rochester Cancer Center
Rochester, New York, United States, 14642
United States, Ohio
Ireland Cancer Center
Cleveland, Ohio, United States, 44106-5065
United States, Tennessee
Vanderbilt Cancer Center
Nashville, Tennessee, United States, 37232-6838
Veterans Affairs Medical Center - Nashville
Nashville, Tennessee, United States, 37212
United States, Wisconsin
CCOP - Marshfield Medical Research and Education Foundation
Marshfield, Wisconsin, United States, 54449
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Veterans Affairs Medical Center - Milwaukee (Zablocki)
Milwaukee, Wisconsin, United States, 53295
Sponsors and Collaborators
Eastern Cooperative Oncology Group
Investigators
Study Chair: Morie A. Gertz, MD Mayo Clinic
  More Information

Additional Information:
Publications:
Study ID Numbers: CDR0000066334, E-4A97
Study First Received: November 1, 1999
Last Updated: February 6, 2009
ClinicalTrials.gov Identifier: NCT00003353     History of Changes
Health Authority: United States: Federal Government

Keywords provided by National Cancer Institute (NCI):
primary systemic amyloidosis

Study placed in the following topic categories:
Melphalan
Metabolic Diseases
Immunoproliferative Disorders
Immunologic Factors
Blood Protein Disorders
Hematologic Diseases
Blood Coagulation Disorders
Vascular Diseases
Paraproteinemias
Hemostatic Disorders
Immunosuppressive Agents
Multiple Myeloma
Amyloidosis
Hemorrhagic Disorders
Antineoplastic Agents, Alkylating
Lymphoproliferative Disorders
Alkylating Agents
Metabolic Disorder
Neoplasms, Plasma Cell

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

ClinicalTrials.gov processed this record on September 01, 2009