Expired Study
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Boston, Massachusetts 02118


Purpose:

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.


Study summary:

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.


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 - 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 is Available At:


Original ID:

CDR0000347379


NCT ID:

NCT00075608


Secondary ID:

BUMC-2001-0156


Study Acronym:


Brief Title:

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


Official Title:

Phase II Trial of Second Autologous Transplantation in AL Amyloidosis


Overall Status:

Completed


Study Phase:

Phase 2


Genders:

Both


Minimum Age:

18 Years


Maximum Age:

65 Years


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

Boston Medical Center


Oversight Authority:

United States: Food and Drug Administration


Reasons Why Stopped:


Study Type:

Interventional


Study Design:

Intervention Model: Single Group Assignment, Mask


Number of Arms:

1


Number of Groups:

0


Total Enrollment:

12


Enrollment Type:

Actual


Overall Contact Information

Official Name:Karen Quillen, MD
Principal Investigator
Boston Medical Center

Study Dates

Start Date:August 2001
Completion Date:October 2011
Completion Type:Actual
Primary Completion Date:October 2011
Primary Completion Type:Actual
Verification Date:December 2011
Last Changed Date:December 9, 2011
First Received Date:January 9, 2004

Study Outcomes

Outcome Type:Primary Outcome
Measure:Evaluate immune reconstitution
Time Frame:3 months after treatment and annually
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Response and durability of response
Time Frame:3 months after treatment and annually
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Feasibility and tolerability
Time Frame:3 months after treatment and annually
Safety Issues:True

Study Interventions

Intervention Type:Biological
Name:filgrastim
Description:16mcg/kg IV daily beginning three days prior to SCC through last day of SCC
Other Name:G-CSF
Intervention Type:Drug
Name:melphalan
Description:140-200 mcg/kg IV over two days
Other Name:alkeran
Intervention Type:Procedure
Name:autologous bone marrow transplantation
Description:infusion of previously collected stem cells on Day 0
Intervention Type:Procedure
Name:peripheral blood stem cell transplantation
Description:infusion of previously collected stem cells on Day 0

Study Arms

There are no available Study Arms

Study Agencies

Agency Class:Other
Agency Type:Lead Sponsor
Agency Name:Boston Medical Center

Sample and Retention Information

There are no available Sample and Retention Information

Study References

There are no available Study References

Data Source: ClinicalTrials.gov

Date Processed: November 18, 2019

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