NCT00052884

Amifostine and Melphalan in Treating Patients With Primary Systemic Amyloidosis Who Are Undergoing Peripheral Stem Cell Transplantation

A Phase I Study of Amifostine Followed by High-Dose Escalation of Melphalan With Stem Cell Reconstitution for Patients With Primary Systemic Amyloidosis

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Eastern Cooperative Oncology Group · Network
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a peripheral stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher dose of chemotherapy to be given so that more plasma cells are killed. Giving a chemoprotective drug such as amifostine may protect kidney cells from the side effects of chemotherapy. PURPOSE: This phase I trial is studying the side effects and best dose of melphalan given together with amifostine in treating patients who are undergoing peripheral stem cell transplant for primary systemic amyloidosis.

Detailed description

OBJECTIVES: * Determine the maximum tolerated dose (MTD) of high-dose melphalan administered with amifostine in patients with primary systemic amyloidosis undergoing autologous peripheral blood stem cell transplantation. * Determine the toxicity of high-dose melphalan when administered at the MTD in these patients. * Determine the response rate in patients treated with this regimen. OUTLINE: This is a nonrandomized, multicenter, dose-escalation study of melphalan. Patients receive filgrastim (G-CSF) subcutaneously once daily until peripheral blood stem cell (PBSC) collection is complete. Apheresis begins on day 5 of G-CSF administration and continues until the target number of PBSCs are collected. Within 6 weeks of PBSC collection, patients receive amifostine IV over 5 minutes on days -2 and -1 and high-dose melphalan IV over 30-60 minutes on day -1. Patients undergo autologous PBSC infusion on day 0. Cohorts of 3-6 patients receive escalating doses of melphalan until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which at least 2 of 3 or 2 of 6 patients experience dose-limiting toxicity. Once the MTD is determined, an additional 10 patients are treated at that dose. Patients are followed approximately 3 months following transplantation, then every 6 months for 5 years. PROJECTED ACCRUAL: A total of 3-46 patients will be accrued for this study within 2.3 years.

Conditions

• Drug/Agent Toxicity by Tissue/Organ
• Multiple Myeloma and Plasma Cell Neoplasm

Interventions

Type	Name	Description
BIOLOGICAL	filgrastim
DRUG	amifostine trihydrate
DRUG	melphalan
PROCEDURE	bone marrow ablation with stem cell support
PROCEDURE	peripheral blood stem cell transplantation

Timeline

• Start date: 2004-01-22
• Primary completion: 2007-09-01
• Completion: 2011-03-01
• First posted: 2003-01-27
• Last updated: 2023-06-18

Locations

16 sites across 1 country: United States