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

Multiple Myeloma and Plasma Cell Neoplasm Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00003353
• Other study ID #: CDR0000066334
• Secondary ID: E-4A97
• Status: Completed
• Phase: Phase 2
• Start date: November 16, 1998

Study information

• Verified date: June 2023
• Source: Eastern Cooperative Oncology Group
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Est. primary completion date: December 2002
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Amyloidosis
• Immunoglobulin Light-chain Amyloidosis
• Multiple Myeloma
• Multiple Myeloma and Plasma Cell Neoplasm
• Neoplasms, Plasma Cell
• Plasmacytoma

Intervention

Biological:
• filgrastim

Drug:
• melphalan

Procedure:
• bone marrow ablation with stem cell support

• peripheral blood stem cell transplantation

Locations

Country	Name	City	State
United States	New England Medical Center Hospital	Boston	Massachusetts
United States	Ireland Cancer Center	Cleveland	Ohio
United States	CCOP - Evanston	Evanston	Illinois
United States	Indiana University Cancer Center	Indianapolis	Indiana
United States	Veterans Affairs Medical Center - Indianapolis (Roudebush)	Indianapolis	Indiana
United States	CCOP - Marshfield Medical Research and Education Foundation	Marshfield	Wisconsin
United States	Sylvester Cancer Center, University of Miami	Miami	Florida
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	Veterans Affairs Medical Center - Milwaukee (Zablocki)	Milwaukee	Wisconsin
United States	Vanderbilt Cancer Center	Nashville	Tennessee
United States	Veterans Affairs Medical Center - Nashville	Nashville	Tennessee
United States	CCOP - Illinois Oncology Research Association	Peoria	Illinois
United States	Mayo Clinic Cancer Center	Rochester	Minnesota
United States	University of Rochester Cancer Center	Rochester	New York
United States	CCOP - Metro-Minnesota	Saint Louis Park	Minnesota
United States	CCOP - Scottsdale Oncology Program	Scottsdale	Arizona
United States	H. Lee Moffitt Cancer Center and Research Institute	Tampa	Florida

Sponsors (2)

Lead Sponsor	Collaborator
Eastern Cooperative Oncology Group	National Cancer Institute (NCI)

Country where clinical trial is conducted

United States, 

References & Publications (1)

Gertz MA, Blood E, Vesole DH, Abonour R, Lazarus HM, Greipp PR. A multicenter phase 2 trial of stem cell transplantation for immunoglobulin light-chain amyloidosis (E4A97): an Eastern Cooperative Oncology Group Study. Bone Marrow Transplant. 2004 Jul;34(2 — View Citation