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

Sponsor

Herbert Irving Comprehensive Cancer Center (Other)

Overall Status

Completed

CT.gov ID

NCT00017680

Collaborator

(none)

Enrollment

Location

Duration (Months)

0.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Determine the toxicity of this regimen in these patients.

Condition or Disease	Intervention/Treatment	Phase
Amyloidosis	Drug: Melphalan Procedure: Autologous Stem Cell Transplantation	Phase 2

Detailed 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.

Study Design

Study Type:

Interventional

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Jul 1, 1999

Actual Primary Completion Date :

Apr 1, 2004

Actual Study Completion Date :

Apr 1, 2004

Outcome Measures

Primary Outcome Measures

Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction []

Secondary Outcome Measures

Toxicity of high dose chemotherapy regimen []

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 70 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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