High-Dose Melphalan Plus Peripheral Stem Cell Transplantation in Treating Patients With Primary Systemic Amyloidosis

This study has been completed.
Sponsor:
Collaborator:
Information provided by:
Eastern Cooperative Oncology Group
ClinicalTrials.gov Identifier:
NCT00003353
First received: November 1, 1999
Last updated: August 22, 2013
Last verified: August 2013
  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 2

Study Type: Interventional
Study Design: Primary Purpose: 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 Eastern Cooperative Oncology Group:

Study Start Date: July 1998
Primary Completion Date: December 2002 (Final data collection date for primary outcome measure)
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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
Sylvester Cancer Center, University of Miami
Miami, Florida, United States, 33136
H. Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, United States, 33612
United States, Illinois
CCOP - Evanston
Evanston, Illinois, United States, 60201
CCOP - Illinois Oncology Research Association
Peoria, Illinois, United States, 61602
United States, Indiana
Veterans Affairs Medical Center - Indianapolis (Roudebush)
Indianapolis, Indiana, United States, 46202
Indiana University Cancer Center
Indianapolis, Indiana, United States, 46202-5265
United States, Massachusetts
New England Medical Center Hospital
Boston, Massachusetts, United States, 02111
United States, Minnesota
Mayo Clinic Cancer Center
Rochester, Minnesota, United States, 55905
CCOP - Metro-Minnesota
Saint Louis Park, Minnesota, United States, 55416
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:
ClinicalTrials.gov Identifier: NCT00003353     History of Changes
Other Study ID Numbers: CDR0000066334, E-4A97
Study First Received: November 1, 1999
Last Updated: August 22, 2013
Health Authority: United States: Federal Government

Keywords provided by Eastern Cooperative Oncology Group:
primary systemic amyloidosis

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

ClinicalTrials.gov processed this record on October 21, 2014