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

Herbert Irving Comprehensive Cancer Center

Status and phase: Completed; Phase 2

Conditions: Amyloidosis

Treatments: Drug: Melphalan; Procedure: Autologous Stem Cell Transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT00017680

CPMC-CAMP-009A

CPMC-IRB-9041

199/15927

Details and patient eligibility

About

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.

Full description

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.

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)