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Tandem Autologous Stem Cell Transplantation in Treating Patients With Primary Systemic (AL) Amyloidosis

Primary Purpose

Multiple Myeloma

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
filgrastim
melphalan
autologous peripheral blood stem cell transplantation
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, meeting 1 of the following criteria: Plasma cell dyscrasia, evidenced by 1 of the following: Monoclonal protein in the serum or urine by immunofixation electrophoresis Plasmacytosis of the bone marrow with monoclonal staining for kappa or lambda light chain isotype Macroglossia with at least 1 other site having biopsy proven amyloidosis and absence of a mutant transthyretin is ruled out PATIENT CHARACTERISTICS: Age 18 to 65 Performance status SWOG 0-2 Life expectancy At least 1 year Hematopoietic Not specified Hepatic Not specified Renal Not specified Cardiovascular LVEF ≥ 45% by MUGA or echocardiogram Pulmonary DLCO ≥ 50% Other Not pregnant or nursing Fertile patients must use effective contraception Able to tolerate 2 courses of high-dose therapy HIV negative PRIOR CONCURRENT THERAPY: Biologic therapy Not specified Chemotherapy Prior alkylating agent chemotherapy allowed provided there is no morphologic or cytogenetic evidence of myelodysplastic syndromes Prior total cumulative oral melphalan dose < 300 mg Endocrine therapy Not specified Radiotherapy Not specified Surgery Not specified Other At least 4 weeks since prior cytotoxic therapy and recovered Exclusion Criteria: No senile, secondary, localized, dialysis-related, or familial amyloidosis No overt multiple myeloma (e.g., greater than 30% bone marrow plasmacytosis, extensive [more than 2] lytic lesions, hypercalcemia) Cardiovascular No myocardial infarction within the past 6 months No congestive heart failure No arrhythmia refractory to therapy No evidence of symptomatic transient ischemic attacks or strokes No other malignancy within the past 5 years except adequately treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, or adequately treated stage I or II cancer currently in complete remission

Sites / Locations

  • Cancer Research Center at Boston Medical Center

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

tandem transplant

Arm Description

Drug: filgrastim 16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection Drug: melphalan 200 mg/kg over 2 days Procedure/Surgery: autologous peripheral blood stem cell transplantation autologous peripheral blood stem cell transplantation

Outcomes

Primary Outcome Measures

safety

Secondary Outcome Measures

Efficacy

Full Information

First Posted
January 9, 2004
Last Updated
September 15, 2020
Sponsor
Boston Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT00075621
Brief Title
Tandem Autologous Stem Cell Transplantation in Treating Patients With Primary Systemic (AL) Amyloidosis
Official Title
A Phase II Trial of Tandem Transplantation in AL Amyloidosis
Study Type
Interventional

2. Study Status

Record Verification Date
September 2020
Overall Recruitment Status
Completed
Study Start Date
August 2000 (Actual)
Primary Completion Date
June 2005 (Actual)
Study Completion Date
September 4, 2020 (Actual)

3. Sponsor/Collaborators

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

4. Oversight

Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
RATIONALE: Autologous stem cell transplantation may be effective treatment for primary systemic (AL) amyloidosis. PURPOSE: This phase II trial is studying how well tandem (two) autologous stem cell transplantation works in treating patients with primary systemic (AL) amyloidosis.
Detailed Description
OBJECTIVES: Determine the tolerability of tandem autologous stem cell transplantation in patients with AL amyloidosis. Determine whether this regimen can convert a hematologic non-complete response (CR) to CR in these patients. Determine the overall survival of patients treated with this regimen. OUTLINE: First transplantation: Patients receive filgrastim (G-CSF) subcutaneously once daily beginning 3 days before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection. Patients may undergo bone marrow harvest if an inadequate number of peripheral blood stem cells are collected. Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2. Patients undergo autologous stem cell transplantation (ASCT) on day 0. Second transplantation: Within 6-12 months after the first ASCT, patients not achieving a complete response receive high-dose melphalan IV over 20 minutes on days -3 and -2 and a second ASCT on day 0. Treatment continues in the absence of unacceptable toxicity. Patients are followed at 3 and 6 months, 1 year, and then annually thereafter. PROJECTED ACCRUAL: A total of 62 patients will be accrued for this study within 2-3 years.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Multiple Myeloma
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
62 (Actual)

8. Arms, Groups, and Interventions

Arm Title
tandem transplant
Arm Type
Other
Arm Description
Drug: filgrastim 16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection Drug: melphalan 200 mg/kg over 2 days Procedure/Surgery: autologous peripheral blood stem cell transplantation autologous peripheral blood stem cell transplantation
Intervention Type
Drug
Intervention Name(s)
filgrastim
Intervention Description
16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection
Intervention Type
Drug
Intervention Name(s)
melphalan
Other Intervention Name(s)
alkeran
Intervention Description
200 mg/kg over 2 days
Intervention Type
Procedure
Intervention Name(s)
autologous peripheral blood stem cell transplantation
Intervention Description
autologous peripheral blood stem cell transplantation
Primary Outcome Measure Information:
Title
safety
Time Frame
100 days, 6 months, and annual
Secondary Outcome Measure Information:
Title
Efficacy
Time Frame
one year

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, meeting 1 of the following criteria: Plasma cell dyscrasia, evidenced by 1 of the following: Monoclonal protein in the serum or urine by immunofixation electrophoresis Plasmacytosis of the bone marrow with monoclonal staining for kappa or lambda light chain isotype Macroglossia with at least 1 other site having biopsy proven amyloidosis and absence of a mutant transthyretin is ruled out PATIENT CHARACTERISTICS: Age 18 to 65 Performance status SWOG 0-2 Life expectancy At least 1 year Hematopoietic Not specified Hepatic Not specified Renal Not specified Cardiovascular LVEF ≥ 45% by MUGA or echocardiogram Pulmonary DLCO ≥ 50% Other Not pregnant or nursing Fertile patients must use effective contraception Able to tolerate 2 courses of high-dose therapy HIV negative PRIOR CONCURRENT THERAPY: Biologic therapy Not specified Chemotherapy Prior alkylating agent chemotherapy allowed provided there is no morphologic or cytogenetic evidence of myelodysplastic syndromes Prior total cumulative oral melphalan dose < 300 mg Endocrine therapy Not specified Radiotherapy Not specified Surgery Not specified Other At least 4 weeks since prior cytotoxic therapy and recovered Exclusion Criteria: No senile, secondary, localized, dialysis-related, or familial amyloidosis No overt multiple myeloma (e.g., greater than 30% bone marrow plasmacytosis, extensive [more than 2] lytic lesions, hypercalcemia) Cardiovascular No myocardial infarction within the past 6 months No congestive heart failure No arrhythmia refractory to therapy No evidence of symptomatic transient ischemic attacks or strokes No other malignancy within the past 5 years except adequately treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, or adequately treated stage I or II cancer currently in complete remission
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Vaishali Sanchorawala, MD
Organizational Affiliation
Boston Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cancer Research Center at Boston Medical 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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Tandem Autologous Stem Cell Transplantation in Treating Patients With Primary Systemic (AL) Amyloidosis

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