Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Boston Medical Center Identifier:
First received: January 9, 2004
Last updated: January 29, 2015
Last verified: July 2013

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
Drug: filgrastim
Drug: melphalan
Procedure: autologous peripheral blood stem cell transplantation
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II Trial of Tandem Transplantation in AL Amyloidosis

Resource links provided by NLM:

Further study details as provided by Boston Medical Center:

Primary Outcome Measures:
  • safety [ Time Frame: 100 days, 6 months, and annual ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Efficacy [ Time Frame: one year ] [ Designated as safety issue: No ]

Enrollment: 62
Study Start Date: August 2000
Estimated Study Completion Date: May 2016
Primary Completion Date: June 2005 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

Detailed Description:


  • 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.


  • 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.


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

Inclusion Criteria:


  • 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



  • 18 to 65

Performance status

  • SWOG 0-2

Life expectancy

  • At least 1 year


  • Not specified


  • Not specified


  • Not specified


  • LVEF ≥ 45% by MUGA or echocardiogram


  • DLCO ≥ 50%


  • Not pregnant or nursing
  • Fertile patients must use effective contraception
  • Able to tolerate 2 courses of high-dose therapy
  • HIV negative


Biologic therapy

  • Not specified


  • 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


  • Not specified


  • Not specified


  • 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)


  • 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
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Please refer to this study by its identifier: NCT00075621

United States, Massachusetts
Cancer Research Center at Boston Medical Center
Boston, Massachusetts, United States, 02118
Sponsors and Collaborators
Boston Medical Center
Principal Investigator: Vaishali Sanchorawala, MD Boston Medical Center
  More Information

Additional Information:
No publications provided

Responsible Party: Boston Medical Center Identifier: NCT00075621     History of Changes
Other Study ID Numbers: CDR0000347381, BUMC-2000-0279
Study First Received: January 9, 2004
Last Updated: January 29, 2015
Health Authority: United States: Federal Government

Keywords provided by Boston Medical Center:
primary systemic amyloidosis processed this record on February 27, 2015