We updated the design of this site on December 18, 2017. Learn more.
ClinicalTrials.gov
ClinicalTrials.gov Menu
IMPORTANT: Due to the lapse in government funding, the information on this web site may not be up to date, transactions submitted via the web site may not be processed, and the agency may not be able to respond to inquiries until appropriations are enacted. Updates regarding government operating status and resumption of normal operations can be found at opm.gov.

Tandem Autologous Stem Cell Transplantation in Treating Patients With Primary Systemic (AL) Amyloidosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00075621
Recruitment Status : Active, not recruiting
First Posted : January 12, 2004
Last Update Posted : April 10, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:

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 or disease Intervention/treatment Phase
Multiple Myeloma Drug: filgrastim Drug: melphalan Procedure: autologous peripheral blood stem cell transplantation Phase 2

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.


Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 62 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Trial of Tandem Transplantation in AL Amyloidosis
Study Start Date : August 2000
Primary Completion Date : June 2005
Estimated Study Completion Date : May 2018


Arms and Interventions

Arm Intervention/treatment
tandem transplant

Drug: filgrastim 16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection

Drug: melphalan 200 mg/kg over 2 days

Procedure/Surgery: autologous peripheral blood stem cell transplantation

autologous peripheral blood stem cell transplantation

Drug: filgrastim
16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection
Drug: melphalan
200 mg/kg over 2 days
Other Name: alkeran
Procedure: autologous peripheral blood stem cell transplantation
autologous peripheral blood stem cell transplantation


Outcome Measures

Primary Outcome Measures :
  1. safety [ Time Frame: 100 days, 6 months, and annual ]

Secondary Outcome Measures :
  1. Efficacy [ Time Frame: one year ]

Eligibility Criteria

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion 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

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

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

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

Exclusion Criteria:

  • 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)

Cardiovascular

  • 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
  • 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
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

Responsible Party: Vaishali Sanchorawala, BMC Faculty, Boston Medical Center
ClinicalTrials.gov Identifier: NCT00075621     History of Changes
Other Study ID Numbers: CDR0000347381
BUMC-2000-0279 ( Other Identifier: BUMC IRB )
First Posted: January 12, 2004    Key Record Dates
Last Update Posted: April 10, 2017
Last Verified: April 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Vaishali Sanchorawala, Boston Medical Center:
primary systemic amyloidosis

Additional relevant MeSH terms:
Multiple Myeloma
Amyloidosis
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Neoplasms
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Proteostasis Deficiencies
Metabolic Diseases
Melphalan
Lenograstim
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Adjuvants, Immunologic