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Phase II Trial of High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis


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

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Trial Information

Phase II Trial of High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis


OBJECTIVES:

- To determine if hematologic responses to high-dose melphalan and autologous stem cell
transplantation increase with addition of bortezomib in the conditioning regimen in
patients with primary systemic amyloidosis.

OUTLINE:

- Autologous stem cell mobilization and collection: Patients receive filgrastim (G-CSF)
to mobilize stem cells, which are then collected.

- Conditioning regimen: Patients receive bortezomib IV on days -6, -3, 1, and 4 and oral
high-dose melphalan on days -2 and -1.

- Stem cell transplantation: Patients undergo autologous stem cell transplantation on day
0.

After completion of study therapy, patients are followed every 6 months for 1 year and
annually thereafter.

Inclusion Criteria


DISEASE CHARACTERISTICS:

- Histologically confirmed primary systemic amyloidosis based on the following
criteria:

- Amyloid light-chain disease

- Deposition of amyloid material by congo red stain showing characteristic green
birefringence

- Monoclonal light chain protein (Bence Jones protein) in the serum or urine,
immunohistochemical studies, or serum free light chain assay

- Evidence of tissue involvement other than carpal tunnel syndrome (i.e., positive
immunohistochemical staining of bone marrow demonstrating clonal plasma cells);
tissue amyloid deposits with anti-kappa or anti-lambda anti-serum; evidence for
a PCD by serum/urine or bone marrow; or overwhelmingly convincing clinical
features (e.g., macroglossia) associated with other systemic manifestations

- No senile, secondary, localized, dialysis-related, or familial amyloidosis

- No overt multiple myeloma (> 30% of bone marrow plasmacytosis, extensive [> 2] lytic
lesions, or hypercalcemia)

PATIENT CHARACTERISTICS:

- SWOG performance status 0-1

- Not pregnant or nursing

- Fertile patients must use effective contraception

- LVEF ≥ 45% by ECHO within the past 60 days

- DLCO ≥ 50%

- No myocardial infarction within the past 6 months, congestive heart failure, or
arrhythmia refractory to therapy

- No prior malignancy except for any of the following:

- Adequately treated basal cell or squamous cell skin cancer

- In situ cervical cancer

- Adequately treated stage I or II cancer currently in complete remission

- Any cancer from which the patient has been disease-free ≥ 5 years

- No advanced (grade 3-4) pre-existing neuropathy

- No HIV positivity

PRIOR CONCURRENT THERAPY:

- Prior chemotherapy with alkylating agent allowed provided there is no morphological
or cytogenetic evidence of myelodysplastic syndromes

- Prior total cumulative dose of oral melphalan < 300 mg

- At least 4 weeks since prior cytotoxic therapy and fully recovered

Type of Study:

Interventional

Study Design:

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Hematologic response (complete and partial)

Outcome Time Frame:

one year

Safety Issue:

No

Principal Investigator

Vaishali Sanchorawala, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Boston Medical Center

Authority:

United States: Food and Drug Administration

Study ID:

CDR0000618857

NCT ID:

NCT00790647

Start Date:

June 2008

Completion Date:

Related Keywords:

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

Name

Location

Boston University Cancer Research CenterBoston, Massachusetts  02118