Second Autologous Stem Cell Transplant in Treating Patients With Persistent or Recurrent Primary Systemic (AL) Amyloidosis

Multiple Myeloma and Plasma Cell Neoplasm Clinical Trial

Official title:

Phase II Trial of Second Autologous Transplantation in AL Amyloidosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00075608
• Other study ID #: CDR0000347379
• Secondary ID: BUMC-2001-0156
• Status: Completed
• Phase: Phase 2
• First received: January 9, 2004
• Last updated: December 9, 2011
• Start date: August 2001
• Est. completion date: October 2011

Study information

• Verified date: December 2011
• Source: Boston Medical Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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 stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more plasma cells are killed. By reducing the number of plasma cells, the disease may progress more slowly.

PURPOSE: This phase II trial is studying how well autologous stem cell transplant works in treating patients with persistent or recurrent primary systemic (AL) amyloidosis.

Description:

OBJECTIVES:

• Determine the feasibility and tolerability of second autologous stem cell transplantation in patients with persistent or recurrent AL amyloidosis.

• Determine the response rate and durability of response in patients treated with this regimen.

• Determine immune reconstitution in patients treated with this regimen.

OUTLINE:

• Mobilization: Patients receive filgrastim (G-CSF) subcutaneously (SC) once daily beginning before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection.

• Preparative regimen: Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2.

• Autologous stem cell transplantation: Autologous stem cells are reinfused on day 0.

Patients are followed at 6 months, 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 19 patients will be accrued for this study within 5-6 years.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: October 2011
• Est. primary completion date: October 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

DISEASE CHARACTERISTICS:

• Histologically confirmed AL amyloidosis

• Persistent or recurrent disease after 1 course of prior high-dose chemotherapy

• Previously treated with autologous stem cell transplantation

• Significant initial improvement in organ function after prior high-dose melphalan, defined by at least 1 of the following:

• Complete hematologic remission (e.g., absence of monoclonal spike by immunofixation in serum and urine AND less then 5% plasma cells in bone marrow with no clonal predominance) OR partial hematologic response (e.g., any decrease in serum or urine monoclonal protein OR decrease in bone marrow plasmacytosis)

• Greater than 50% reduction in proteinuria with preservation of creatinine clearance

• Greater than 50% reduction in alkaline phosphatase OR at least 2 cm decrease in liver size by physical exam

• Subjective neurologic improvement, as confirmed by neurologist

• Cardiac stabilization of disease confirmed by echocardiography defined as less than 2 mm increase in mean wall thickness and/or less than 20 g increase in left ventricular mass

• Improvement in performance status* NOTE: *This criteria alone does not constitute significant improvement in organ function

• No myelodysplastic syndromes

• No abnormal bone marrow cytogenetics

• Prior stem cell yield must have been = 2 x 10^6 CD34+ cells/kg

PATIENT CHARACTERISTICS:

Age

• 18 to 65

Performance status

• SWOG 0-2

Life expectancy

• More than 6 months

Hematopoietic

• See Disease Characteristics

Hepatic

• See Disease Characteristics

Renal

• See Disease Characteristics

Cardiovascular

• See Disease Characteristics

• LVEF = 45% by MUGA or echocardiogram

Pulmonary

• DLCO = 50%

Other

• Not pregnant or nursing

• Fertile patients must use effective contraception

• Acceptable toxicity from first transplantation, confirmed by the transplant team

• HIV negative

• No other concurrent malignancy except treated skin cancer

PRIOR CONCURRENT THERAPY:

Biologic therapy

• See Disease Characteristics

Chemotherapy

• See Disease Characteristics

• No chemotherapy after first transplantation

Endocrine therapy

• Not specified

Radiotherapy

• Not specified

Surgery

• Not specified

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyloidosis
• Multiple Myeloma
• Multiple Myeloma and Plasma Cell Neoplasm
• Neoplasms, Plasma Cell
• Plasmacytoma

Intervention

Biological:
• filgrastim
16mcg/kg IV daily beginning three days prior to SCC through last day of SCC

Drug:
• melphalan
140-200 mcg/kg IV over two days

Procedure:
• autologous bone marrow transplantation
infusion of previously collected stem cells on Day 0
• peripheral blood stem cell transplantation
infusion of previously collected stem cells on Day 0

Locations

Country	Name	City	State
United States	Boston University Cancer Research Center	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Boston Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Feasibility and tolerability		3 months after treatment and annually	Yes
Primary	Response and durability of response		3 months after treatment and annually	No
Primary	Evaluate immune reconstitution		3 months after treatment and annually	No