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

This study has been completed.
Sponsor:
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00017680
First received: June 6, 2001
Last updated: June 6, 2008
Last verified: June 2008
  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 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in

Resource links provided by NLM:


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:
    Drug: Melphalan
    100 mg/m2 twice a day from Days -2 and -1 as induction therapy
    Procedure: Autologous Stem Cell Transplantation
    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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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: Charles Hesdorffer, MD, Columbia University
ClinicalTrials.gov Identifier: NCT00017680     History of Changes
Other Study ID Numbers: 199/15927, CPMC-IRB-9041, CPMC-CAMP-009A
Study First Received: June 6, 2001
Last Updated: June 6, 2008
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:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Melphalan
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antineoplastic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on July 20, 2014