2nd Autologous Stem Cell Transplant in Patients With Persistent/Recurrent (AL) Amyloidosis

NCT ID: NCT00075608

Last Updated: 2017-01-27

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2001-08-31
• Study Completion Date: 2011-10-31

Brief 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.

Detailed 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.

Conditions

Multiple Myeloma; Plasma Cell Neoplasm

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

2nd Stem Cell Transplant

Mobilization with filgrastim autologous stem cell transplantation with melphalan conditioning stem cell infusion

Group Type: EXPERIMENTAL

filgrastim

Intervention Type: BIOLOGICAL

16mcg/kg IV daily beginning three days prior to stem cell collection through last day of stem cell collection

melphalan

Intervention Type: DRUG

140-200 mcg/kg IV over two days

autologous stem cell transplantation

Intervention Type: PROCEDURE

infusion of previously collected stem cells on Day 0

stem cell infusion

Intervention Type: PROCEDURE

infusion of previously collected stem cells on Day 0

Interventions

filgrastim

16mcg/kg IV daily beginning three days prior to stem cell collection through last day of stem cell collection

Intervention Type: BIOLOGICAL

melphalan

140-200 mcg/kg IV over two days

Intervention Type: DRUG

autologous stem cell transplantation

infusion of previously collected stem cells on Day 0

Intervention Type: PROCEDURE

stem cell infusion

infusion of previously collected stem cells on Day 0

Intervention Type: PROCEDURE

Other Intervention Names

G-CSF; alkeran

Eligibility Criteria

Inclusion Criteria

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

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

PATIENT CHARACTERISTICS:

Age

• 18 to 65

Performance status

• Southwest Oncology Group- 0-2

Life expectancy

• More than 6 months

Hematopoietic

• See Disease Characteristics

Hepatic

• See Disease Characteristics

Renal

• See Disease Characteristics

Cardiovascular

• See Disease Characteristics
• Left ventricular ejection fraction ≥ 45% by multiple gated acquisition scan or echocardiogram

Pulmonary

• diffusing capacity of lung for carbon monoxide ≥ 50%

Exclusion Criteria

• No myelodysplastic syndromes
• No abnormal bone marrow cytogenetics

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Boston Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Karen Quillen

Medical Director, Blood Bank

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Karen Quillen, MD

Role: PRINCIPAL_INVESTIGATOR

Boston Medical Center

Locations

Boston University Cancer Research Center

Boston, Massachusetts, United States

Countries

United States

Other Identifiers

H-22603

Identifier Type: OTHER

Identifier Source: secondary_id

CDR0000347379

Identifier Type: -

Identifier Source: org_study_id