Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Trial Contact Information
Registry Information

Alternate Title

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

Basic Trial Information

Phase	Type	Status	Age	Sponsor	Protocol IDs
Phase II	Treatment	Closed	16 to 65	Other	TUHSC-2797 NCI-V96-0951, NCT00002810

Objectives

1. Assess overall and progression-free survival following high-dose melphalan and autologous peripheral blood stem cell transplantation in patients with primary amyloidosis.
2. Evaluate the toxic effects associated with this treatment regimen.
3. Evaluate the function of involved organs, especially the heart, lungs, and nervous system, before and after treatment with this regimen.

Entry Criteria

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

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

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:

• FEV₁ 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

Expected Enrollment

A very small number of patients are expected to be accrued over 5-10 years.

Outcomes

Overall survival
Time to clinical progression of amyloid symptoms

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.

Trial Contact Information

Trial Lead Organizations

Fox Chase Cancer Center CCOP Research Base

Kenneth Mangan, MD, FACP, Protocol chair		Ph: 215-214-3129 Email: mangank@tuhs.temple.edu

Registry Information
Official Title		Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL)
Trial Start Date		1996-05-23
Registered in ClinicalTrials.gov		NCT00002810
Date Submitted to PDQ		1996-09-14
Information Last Verified		2006-05-18

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