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

Multiple Myeloma and Plasma Cell Neoplasm Clinical Trial

Official title:

Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00002810
• Other study ID #: CDR0000064938
• Secondary ID: TUHSC-2797NCI-V9
• Status: Completed
• Phase: Phase 2
• First received: November 1, 1999
• Last updated: September 30, 2010
• Start date: May 1996
• Est. completion date: May 2006

Study information

• Verified date: September 2010
• Source: Temple University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Est. completion date: May 2006
• Est. primary completion date: May 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years to 65 Years

Eligibility

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

Study Design

Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• 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	Fox Chase-Temple Cancer Center CCOP Research Base	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Temple University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall survival			No
Primary	Time to clinical progression of amyloid symptoms			No