Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain Amyloidosis - Full Text View

Sponsor:	Herbert Irving Comprehensive Cancer Center
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00017680

Purpose

OBJECTIVES: I. Determine the response, disease-free survival, and overall survival of patients with primary light chain amyloidosis treated with high-dose melphalan and autologous stem cell transplantation.

II. Determine the toxicity of this regimen in these patients.

Condition	Intervention	Phase
Amyloidosis	Drug: Melphalan Procedure: Autologous Stem Cell Transplantation	Phase II

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Single Group Assignment
Official Title:	Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in

Resource links provided by NLM:

Genetics Home Reference related topics: transthyretin amyloidosis

MedlinePlus related topics: Amyloidosis Cancer

Drug Information available for: Melphalan Sarcolysin Melphalan hydrochloride

U.S. FDA Resources

Further study details as provided by Office of Rare Diseases (ORD):

Primary Outcome Measures:

Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction

Secondary Outcome Measures:

Toxicity of high dose chemotherapy regimen

Estimated Enrollment:	25
Study Start Date:	July 1999
Study Completion Date:	April 2004
Primary Completion Date:	April 2004 (Final data collection date for primary outcome measure)

Intervention Details:

100 mg/m2 twice a day from Days -2 and -1 as induction therapy

Bone marrow and peripheral blood stem cells harvested

Detailed Description:

PROTOCOL OUTLINE: Patients may receive induction chemotherapy before study entry. Patients then receive filgrastim (G-CSF) or another growth factor for 4-6 days as peripheral blood stem cell (PBSC) mobilization. PBSC (or bone marrow) is harvested over 2-3 days.

Patients receive high-dose melphalan IV over 30 minutes twice daily on days -2 and -1. PBSC and/or bone marrow is reinfused on day 0. Patients receive G-CSF beginning on day 0 and continuing until blood counts recover. This course may be repeated 4-12 weeks later.

Patients are followed every 3 months for 1 year and then annually for 5 years.

Eligibility

Ages Eligible for Study:	18 Years to 70 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

PROTOCOL ENTRY CRITERIA:

Disease Characteristics

Histologically confirmed primary amyloidosis
Ineligible for other high priority national or international study

Prior/Concurrent Therapy

Biologic therapy: Concurrent participation in gene therapy trials allowed
Chemotherapy: Prior chemotherapy allowed No other concurrent chemotherapy
Endocrine therapy: No concurrent steroids unless given with amphotericin B, for adrenal failure, or for septic shock No concurrent hormones except for non-disease-related conditions (e.g., insulin for diabetes)
Other: No concurrent barbiturates or acetaminophen Concurrent participation in supportive care trials allowed

Patient Characteristics

Performance status: ECOG 0-3
Hepatic: Bilirubin less than 2 times normal
Renal: Creatinine less than 2.5 mg/dL OR On stable hemodialysis
Pulmonary: DLCO at least 60% predicted OR Clearance by pulmonologist
Other: HIV negative

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00017680

Locations

United States, New York
Herbert Irving Comprehensive Cancer Center
New York, New York, United States, 10032

Sponsors and Collaborators

Herbert Irving Comprehensive Cancer Center

Investigators

Study Chair:

Charles S. Hesdorffer

Herbert Irving Comprehensive Cancer Center

More Information

No publications provided

Responsible Party:	Columbia University ( Charles Hesdorffer, MD )
Study ID Numbers:	199/15927, CPMC-IRB-9041, CPMC-CAMP-009A
Study First Received:	June 6, 2001
Last Updated:	June 6, 2008
ClinicalTrials.gov Identifier:	NCT00017680 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):

amyloidosis
arthritis & connective tissue diseases
genetic diseases and dysmorphic syndromes
hematopoietic/lymphoid cancer

oncologic disorders
plasma cell neoplasm
primary systemic amyloidosis
rare disease

Additional relevant MeSH terms:

Melphalan
Metabolic Diseases
Immunologic Factors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Physiological Effects of Drugs
Immunosuppressive Agents

Pharmacologic Actions
Amyloidosis
Therapeutic Uses
Myeloablative Agonists
Antineoplastic Agents, Alkylating
Alkylating Agents

ClinicalTrials.gov processed this record on November 27, 2009