Tandem Autologous Stem Cell Transplantation in Treating Patients With Primary Systemic (AL) Amyloidosis - Full Text View

Sponsored by:	Boston Medical Center
Information provided by:	National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:	NCT00075621

Purpose

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.

Condition	Intervention	Phase
Multiple Myeloma and Plasma Cell Neoplasm	Drug: filgrastim Drug: melphalan Procedure: autologous bone marrow transplantation Procedure: peripheral blood stem cell transplantation	Phase II

Genetics Home Reference related topics: aceruloplasminemia hemophilia

MedlinePlus related topics: Bone Marrow Transplantation Cancer Multiple Myeloma

Drug Information available for: Filgrastim Melphalan Melphalan hydrochloride Sarcolysin

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label
Official Title:	A Phase II Trial of Tandem Transplantation in AL Amyloidosis

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

Study Start Date:

August 2000

Detailed 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.

Eligibility

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

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00075621

Locations

United States, Massachusetts
Cancer Research Center at Boston Medical Center
Boston, Massachusetts, United States, 02118

Sponsors and Collaborators

Boston Medical Center

Investigators

Principal Investigator:

Vaishali Sanchorawala, MD

Boston Medical Center

More Information

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

Study ID Numbers:	CDR0000347381, BUMC-2000-0279
Study First Received:	January 9, 2004
Last Updated:	July 23, 2008
ClinicalTrials.gov Identifier:	NCT00075621
Health Authority:	United States: Federal Government

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
Vascular Diseases
Paraproteinemias

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

Additional relevant MeSH terms:

Neoplasms
Neoplasms by Histologic Type
Immune System Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on January 13, 2009