Philadelphia, Pennsylvania 19111

  • Multiple Myeloma and Plasma Cell Neoplasm

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 peripheral 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 giving high-dose melphalan together with peripheral stem cell transplant works in treating patients with primary amyloidosis or amyloidosis associated with multiple myeloma.


Study summary:

OBJECTIVES: - Assess overall and progression-free survival following high-dose melphalan and autologous peripheral blood stem cell transplantation in patients with primary amyloidosis. - Evaluate the toxic effects associated with this treatment regimen. - Evaluate the function of involved organs, especially the heart, lungs, and nervous system, before and after treatment with this regimen. OUTLINE: Peripheral blood stem cells (PBSC) are mobilized with granulocyte colony-stimulating factor (G-CSF) for 5 days and then collected by leukapheresis. Patients receive high-dose melphalan on 2 consecutive days, followed by 1 day of rest, then by PBSC transplantation. G-CSF is given from 1 day after transplantation until the neutrophil count is greater than 1,500 for 3 consecutive days. Patients are followed at 100 days and 1 year post-transplant. PROJECTED ACCRUAL: A very small number of patients are expected to be accrued over 5-10 years.


Criteria:

DISEASE CHARACTERISTICS: - Primary amyloidosis diagnosed by appropriate amyloid stains or electromicroscopy of abdominal fat, bone marrow, or other target tissues - Pathology reviewed by Temple University - Amyloidosis secondary to any stage of multiple myeloma allowed provided plasma cell concentration in bone marrow is less than 15% - No amyloidosis secondary to rheumatoid arthritis or chronic infection - No familial amyloidosis PATIENT CHARACTERISTICS: Age: - 16 to 65 Performance status: - Karnofsky 80-100% Hematopoietic: - Not specified Hepatic: - Liver function tests less than twice normal - No active liver disease Renal: - Creatinine clearance greater than 50 mL/min - Nephrotic syndrome allowed Cardiovascular: - Cardiac evaluation required in patients with left ventricular ejection fraction less than 45% by echocardiogram or MUGA - No poorly controlled hypertension Pulmonary: - FEV_1 and DLCO greater than 50% of predicted, or pulmonary evaluation required - No chronic obstructive pulmonary disease Other: - No history of serious coagulopathy, hemorrhage, or bleeding - No active infection - No other serious comorbid disease (e.g., poorly controlled diabetes) - No pregnant women - Adequate contraception required of fertile women PRIOR CONCURRENT THERAPY: Biologic therapy: - Not specified Chemotherapy: - More than 12 monthly cycles of prior alkylating agent chemotherapy discouraged Endocrine therapy: - Corticosteroids discontinued at least 6 weeks prior to transplantation Radiotherapy: - No prior radiotherapy Surgery: - Not specified


Study is Available At:


Original ID:

CDR0000064938


NCT ID:

NCT00002810


Secondary ID:

TUHSC-2797


Study Acronym:


Brief Title:

High-Dose Melphalan Followed by Peripheral Stem Cell Transplant in Treating Patients With Amyloidosis


Official Title:

Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL)


Overall Status:

Completed


Study Phase:

Phase 2


Genders:

Both


Minimum Age:

16 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:

Temple University


Oversight Authority:

United States: Federal Government


Reasons Why Stopped:


Study Type:

Interventional


Study Design:

Allocation: Non-Randomized, Intervention Model:


Number of Arms:

0


Number of Groups:

0


Total Enrollment:

0


Enrollment Type:


Overall Contact Information

Official Name:Kenneth F. Mangan, MD, FACP
Study Chair
Fox Chase Cancer Center

Study Dates

Start Date:May 1996
Completion Date:May 2006
Completion Type:Actual
Primary Completion Date:May 2006
Primary Completion Type:Actual
Verification Date:September 2010
Last Changed Date:September 30, 2010
First Received Date:November 1, 1999

Study Outcomes

Outcome Type:Primary Outcome
Measure:Time to clinical progression of amyloid symptoms
Safety Issues:False
Outcome Type:Primary Outcome
Measure:Overall survival
Safety Issues:False

Study Interventions

Intervention Type:Procedure
Name:peripheral blood stem cell transplantation
Intervention Type:Procedure
Name:bone marrow ablation with stem cell support
Intervention Type:Drug
Name:melphalan
Intervention Type:Biological
Name:filgrastim

Study Arms

There are no available Study Arms

Study Agencies

Agency Class:Other
Agency Type:Lead Sponsor
Agency Name:Temple University

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: July 29, 2021

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