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

Multiple Myeloma Clinical Trial

Official title:

A Phase II Trial of Tandem Transplantation in AL Amyloidosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00075621
• Other study ID #: H-23645
• Status: Completed
• Phase: Phase 2
• Start date: August 2000
• Est. completion date: September 4, 2020

Study information

• Verified date: September 2020
• Source: Boston Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 62
• Est. completion date: September 4, 2020
• Est. primary completion date: June 2005
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Amyloidosis
• Multiple Myeloma

Intervention

Drug:
• filgrastim
16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection
• melphalan
200 mg/kg over 2 days

Procedure:
• autologous peripheral blood stem cell transplantation
autologous peripheral blood stem cell transplantation

Locations

Country	Name	City	State
United States	Cancer Research Center at Boston Medical Center	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Boston Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	safety		100 days, 6 months, and annual
Secondary	Efficacy		one year