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A Phase II Study of High-Dose Melphalan With Hematopoietic Stem Cell Reconstitution for Patients With Primary Systemic Amyloidosis


Phase 2
18 Years
70 Years
Not Enrolling
Both
Multiple Myeloma and Plasma Cell Neoplasm

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Trial Information

A Phase II Study of High-Dose Melphalan With Hematopoietic Stem Cell Reconstitution for Patients With Primary Systemic Amyloidosis


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.

Inclusion 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

Type of Study:

Interventional

Study Design:

Primary Purpose: Treatment

Principal Investigator

Morie A. Gertz, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Mayo Clinic

Authority:

United States: Federal Government

Study ID:

CDR0000066334

NCT ID:

NCT00003353

Start Date:

July 1998

Completion Date:

Related Keywords:

  • Multiple Myeloma and Plasma Cell Neoplasm
  • primary systemic amyloidosis
  • Amyloidosis
  • Neoplasms
  • Multiple Myeloma
  • Neoplasms, Plasma Cell
  • Plasmacytoma

Name

Location

Mayo Clinic Cancer CenterRochester, Minnesota  55905
H. Lee Moffitt Cancer Center and Research InstituteTampa, Florida  33612
Indiana University Cancer CenterIndianapolis, Indiana  46202-5265
University of Rochester Cancer CenterRochester, New York  14642
Ireland Cancer CenterCleveland, Ohio  44106-5065
Sylvester Cancer Center, University of MiamiMiami, Florida  33136
CCOP - Illinois Oncology Research AssociationPeoria, Illinois  61602
Veterans Affairs Medical Center - Indianapolis (Roudebush)Indianapolis, Indiana  46202
New England Medical Center HospitalBoston, Massachusetts  02111
CCOP - Metro-MinnesotaSaint Louis Park, Minnesota  55416
Vanderbilt Cancer CenterNashville, Tennessee  37232-6838
CCOP - Scottsdale Oncology ProgramScottsdale, Arizona  85259-5404
Medical College of WisconsinMilwaukee, Wisconsin  53226
Veterans Affairs Medical Center - Milwaukee (Zablocki)Milwaukee, Wisconsin  53295
CCOP - EvanstonEvanston, Illinois  60201
CCOP - Marshfield Medical Research and Education FoundationMarshfield, Wisconsin  54449
Veterans Affairs Medical Center - NashvilleNashville, Tennessee  37212