Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis - Full Text View

Sponsored by:	Boston Medical Center
Information provided by:	National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:	NCT00790647

Purpose

RATIONALE: Giving melphalan and bortezomib before and after a stem cell transplant stops the growth of abnormal cells by stopping them from dividing or killing them. Giving colony-stimulating factors, such as G-CSF, and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy and monoclonal antibody therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.

PURPOSE: This phase II trial is studying how well giving melphalan together with bortezomib followed by stem cell transplant works in treating patients with primary systemic amyloidosis.

Condition	Intervention	Phase
Multiple Myeloma and Plasma Cell Neoplasm	Drug: bortezomib Drug: filgrastim Drug: melphalan Procedure: autologous hematopoietic stem cell transplantation	Phase II

Genetics Home Reference related topics: aceruloplasminemia hemophilia

MedlinePlus related topics: Cancer Multiple Myeloma

Drug Information available for: Filgrastim Melphalan Bortezomib Melphalan hydrochloride Sarcolysin

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label
Official Title:	Phase II Trial of High-Dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:

Hematologic response (complete and partial) [ Designated as safety issue: No ]

Secondary Outcome Measures:

Tolerability [ Designated as safety issue: Yes ]
Survival at 1 and 2 years [ Designated as safety issue: No ]

Estimated Enrollment:	10
Study Start Date:	June 2008
Estimated Primary Completion Date:	December 2009 (Final data collection date for primary outcome measure)

Detailed Description:

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.

Eligibility

Ages Eligible for Study:	18 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00790647

Locations

United States, Massachusetts
Boston University Cancer Research Center	Recruiting
Boston, Massachusetts, United States, 02118
Contact: Clinical Trials Office - Boston University Cancer Research Cen 617-353-7571

Sponsors and Collaborators

Boston Medical Center

Investigators

Principal Investigator:	Vaishali Sanchorawala, MD	Boston Medical Center
Investigator:	David C. Seldin, MD, PhD	Boston Medical Center

More Information

Clinical trial summary from the National Cancer Institute's PDQ® database This link exits the ClinicalTrials.gov site

Responsible Party:	Boston University Cancer Research Center ( Vaishali Sanchorawala )
Study ID Numbers:	CDR0000618857, BUMC-H-27277
Study First Received:	November 12, 2008
Last Updated:	November 13, 2008
ClinicalTrials.gov Identifier:	NCT00790647
Health Authority:	Unspecified

Keywords provided by National Cancer Institute (NCI):

primary systemic amyloidosis

Study placed in the following topic categories:

Melphalan
Metabolic Diseases
Immunoproliferative Disorders
Blood Protein Disorders
Hematologic Diseases
Primary Amyloidosis
Blood Coagulation Disorders
Bortezomib
Vascular Diseases

Paraproteinemias
Hemostatic Disorders
Multiple Myeloma
Amyloidosis
Hemorrhagic Disorders
Multiple myeloma
Metabolic disorder
Lymphoproliferative Disorders
Neoplasms, Plasma Cell

Additional relevant MeSH terms:

Neoplasms by Histologic Type
Molecular Mechanisms of Pharmacological Action
Immune System Diseases
Immunologic Factors
Antineoplastic Agents
Physiological Effects of Drugs
Enzyme Inhibitors
Immunosuppressive Agents

Pharmacologic Actions
Protease Inhibitors
Neoplasms
Therapeutic Uses
Myeloablative Agonists
Cardiovascular Diseases
Antineoplastic Agents, Alkylating
Alkylating Agents

ClinicalTrials.gov processed this record on January 16, 2009