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

Amyloidosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00017680
• Other study ID #: 199/15927
• Secondary ID: CPMC-IRB-9041CPM
• Status: Completed
• Phase: Phase 2
• First received: June 6, 2001
• Last updated: June 6, 2008
• Start date: July 1999
• Est. completion date: April 2004

Study information

• Verified date: June 2008
• Source: Office of Rare Diseases (ORD)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial 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.

II. Determine the toxicity of this regimen in these patients.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: April 2004
• Est. primary completion date: April 2004
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 70 Years

Eligibility

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

Study Design

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

Related Conditions & MeSH terms

• Amyloidosis

Intervention

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

Locations

Country	Name	City	State
United States	Herbert Irving Comprehensive Cancer Center	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Herbert Irving Comprehensive Cancer Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction
Secondary	Toxicity of high dose chemotherapy regimen