Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain Amyloidosis

Study Overview
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Brief Summary
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OBJECTIVES: I. Determine the response, disease-free survival, and overall survival of patients with primary light chain amyloidosis treated with high-dose melphalan and autologous stem cell transplantation. II. Determine the toxicity of this regimen in these patients.

Detailed Description
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PROTOCOL OUTLINE: Patients may receive induction chemotherapy before study entry. Patients then receive filgrastim (G-CSF) or another growth factor for 4-6 days as peripheral blood stem cell (PBSC) mobilization. PBSC (or bone marrow) is harvested over 2-3 days. Patients receive high-dose melphalan IV over 30 minutes twice daily on days -2 and -1. PBSC and/or bone marrow is reinfused on day 0. Patients receive G-CSF beginning on day 0 and continuing until blood counts recover. This course may be repeated 4-12 weeks later. Patients are followed every 3 months for 1 year and then annually for 5 years.

Official Title
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Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in

Conditions
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Amyloidosis

Intervention / Treatment
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* Drug: Melphalan
* Procedure: Autologous Stem Cell Transplantation


Participation Criteria
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Eligibility Criteria
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PROTOCOL ENTRY CRITERIA: Disease Characteristics Histologically confirmed primary amyloidosis Ineligible for other high priority national or international study Prior/Concurrent Therapy Biologic therapy: Concurrent participation in gene therapy trials allowed Chemotherapy: Prior chemotherapy allowed No other concurrent chemotherapy Endocrine therapy: No concurrent steroids unless given with amphotericin B, for adrenal failure, or for septic shock No concurrent hormones except for non-disease-related conditions (e.g., insulin for diabetes) Other: No concurrent barbiturates or acetaminophen Concurrent participation in supportive care trials allowed Patient Characteristics Performance status: ECOG 0-3 Hepatic: Bilirubin less than 2 times normal Renal: Creatinine less than 2.5 mg/dL OR On stable hemodialysis Pulmonary: DLCO at least 60% predicted OR Clearance by pulmonologist Other: HIV negative

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Melphalan|100 mg/m2 twice a day from Days -2 and -1 as induction therapy|
|Procedure: Autologous Stem Cell Transplantation|Bone marrow and peripheral blood stem cells harvested|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Toxicity of high dose chemotherapy regimen |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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amyloidosis, arthritis & connective tissue diseases, genetic diseases and dysmorphic syndromes, hematopoietic/lymphoid cancer, oncologic disorders, plasma cell neoplasm, primary systemic amyloidosis, rare disease