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

Primary Purpose

Multiple Myeloma, Plasma Cell Neoplasm

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
filgrastim
melphalan
autologous stem cell transplantation
stem cell infusion
Sponsored by
Boston Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Multiple Myeloma focused on measuring primary systemic amyloidosis

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Boston University Cancer Research Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

2nd Stem Cell Transplant

Arm Description

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

Outcomes

Primary Outcome Measures

Feasibility and Tolerability
Feasibility and tolerability will be evaluated based on participants completing second transplant with tolerable adverse events
Response and Durability of Response
Response and durability of response will be based on hematologic Complete Response or Partial Response and date of relapse or death
Evaluate Immune Reconstitution
Evaluate immune reconstitution based on time to engraftment

Secondary Outcome Measures

Full Information

First Posted
January 9, 2004
Last Updated
December 2, 2016
Sponsor
Boston Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT00075608
Brief Title
2nd Autologous Stem Cell Transplant in Patients With Persistent/Recurrent (AL) Amyloidosis
Official Title
Phase II Trial of Second Autologous Transplantation in AL Amyloidosis
Study Type
Interventional

2. Study Status

Record Verification Date
December 2016
Overall Recruitment Status
Terminated
Why Stopped
poor accrual
Study Start Date
August 2001 (undefined)
Primary Completion Date
October 2011 (Actual)
Study Completion Date
October 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Boston Medical Center

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Multiple Myeloma, Plasma Cell Neoplasm
Keywords
primary systemic amyloidosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
2nd Stem Cell Transplant
Arm Type
Experimental
Arm Description
Mobilization with filgrastim autologous stem cell transplantation with melphalan conditioning stem cell infusion
Intervention Type
Biological
Intervention Name(s)
filgrastim
Other Intervention Name(s)
G-CSF
Intervention Description
16mcg/kg IV daily beginning three days prior to stem cell collection through last day of stem cell collection
Intervention Type
Drug
Intervention Name(s)
melphalan
Other Intervention Name(s)
alkeran
Intervention Description
140-200 mcg/kg IV over two days
Intervention Type
Procedure
Intervention Name(s)
autologous stem cell transplantation
Intervention Description
infusion of previously collected stem cells on Day 0
Intervention Type
Procedure
Intervention Name(s)
stem cell infusion
Intervention Description
infusion of previously collected stem cells on Day 0
Primary Outcome Measure Information:
Title
Feasibility and Tolerability
Description
Feasibility and tolerability will be evaluated based on participants completing second transplant with tolerable adverse events
Time Frame
3 months after treatment and annually
Title
Response and Durability of Response
Description
Response and durability of response will be based on hematologic Complete Response or Partial Response and date of relapse or death
Time Frame
3 months after treatment and annually
Title
Evaluate Immune Reconstitution
Description
Evaluate immune reconstitution based on time to engraftment
Time Frame
3 months after treatment and annually

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Karen Quillen, MD
Organizational Affiliation
Boston Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Boston University Cancer Research Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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

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