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A Phase II Trial of Tandem Transplantation in AL Amyloidosis


Phase 2
18 Years
65 Years
Open (Enrolling)
Both
Multiple Myeloma and Plasma Cell Neoplasm

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Trial Information

A Phase II Trial of Tandem Transplantation in AL Amyloidosis


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.

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

- 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)

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

- 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

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

- 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

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

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

safety

Outcome Time Frame:

100 days, 6 months, and annual

Safety Issue:

Yes

Principal Investigator

Vaishali Sanchorawala, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Boston Medical Center

Authority:

United States: Federal Government

Study ID:

CDR0000347381

NCT ID:

NCT00075621

Start Date:

August 2000

Completion Date:

May 2015

Related Keywords:

  • Multiple Myeloma and Plasma Cell Neoplasm
  • primary systemic amyloidosis
  • Amyloidosis
  • Neoplasms
  • Multiple Myeloma
  • Neoplasms, Plasma Cell
  • Plasmacytoma

Name

Location

Cancer Research Center at Boston Medical CenterBoston, Massachusetts  02118