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2nd Autologous Stem Cell Transplant in Patients With Persistent/Recurrent (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. Identifier: NCT00075608
Recruitment Status : Terminated (poor accrual)
First Posted : January 12, 2004
Results First Posted : January 27, 2017
Last Update Posted : January 27, 2017
Information provided by (Responsible Party):
Karen Quillen, Boston Medical Center

Brief Summary:

RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more plasma cells are killed. By reducing the number of plasma cells, the disease may progress more slowly.

PURPOSE: This phase II trial is studying how well autologous stem cell transplant works in treating patients with persistent or recurrent primary systemic (AL) amyloidosis.

Condition or disease Intervention/treatment Phase
Multiple Myeloma Plasma Cell Neoplasm Biological: filgrastim Drug: melphalan Procedure: autologous stem cell transplantation Procedure: stem cell infusion Phase 2

Detailed Description:


  • Determine the feasibility and tolerability of second autologous stem cell transplantation in patients with persistent or recurrent AL amyloidosis.
  • Determine the response rate and durability of response in patients treated with this regimen.
  • Determine immune reconstitution in patients treated with this regimen.


  • Mobilization: Patients receive filgrastim (G-CSF) subcutaneously (SC) once daily beginning before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection.
  • Preparative regimen: Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2.
  • Autologous stem cell transplantation: Autologous stem cells are reinfused on day 0.

Patients are followed at 6 months, 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 19 patients will be accrued for this study within 5-6 years.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Trial of Second Autologous Transplantation in AL Amyloidosis
Study Start Date : August 2001
Actual Primary Completion Date : October 2011
Actual Study Completion Date : October 2011

Arm Intervention/treatment
Experimental: 2nd Stem Cell Transplant
Mobilization with filgrastim autologous stem cell transplantation with melphalan conditioning stem cell infusion
Biological: filgrastim
16mcg/kg IV daily beginning three days prior to stem cell collection through last day of stem cell collection
Other Name: G-CSF

Drug: melphalan
140-200 mcg/kg IV over two days
Other Name: alkeran

Procedure: autologous stem cell transplantation
infusion of previously collected stem cells on Day 0

Procedure: stem cell infusion
infusion of previously collected stem cells on Day 0

Primary Outcome Measures :
  1. Feasibility and Tolerability [ Time Frame: 3 months after treatment and annually ]
    Feasibility and tolerability will be evaluated based on participants completing second transplant with tolerable adverse events

  2. Response and Durability of Response [ Time Frame: 3 months after treatment and annually ]
    Response and durability of response will be based on hematologic Complete Response or Partial Response and date of relapse or death

  3. Evaluate Immune Reconstitution [ Time Frame: 3 months after treatment and annually ]
    Evaluate immune reconstitution based on time to engraftment

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

Inclusion criteria:


  • Histologically confirmed AL amyloidosis

    • Persistent or recurrent disease after 1 course of prior high-dose chemotherapy
  • Previously treated with autologous stem cell transplantation
  • Significant initial improvement in organ function after prior high-dose melphalan, defined by at least 1 of the following:

    • Complete hematologic remission (e.g., absence of monoclonal spike by immunofixation in serum and urine AND less then 5% plasma cells in bone marrow with no clonal predominance) OR partial hematologic response (e.g., any decrease in serum or urine monoclonal protein OR decrease in bone marrow plasmacytosis)
    • Greater than 50% reduction in proteinuria with preservation of creatinine clearance
    • Greater than 50% reduction in alkaline phosphatase OR at least 2 cm decrease in liver size by physical exam
    • Subjective neurologic improvement, as confirmed by neurologist
    • Cardiac stabilization of disease confirmed by echocardiography defined as less than 2 mm increase in mean wall thickness and/or less than 20 g increase in left ventricular mass
    • Improvement in performance status* NOTE: *This criteria alone does not constitute significant improvement in organ function
  • Prior stem cell yield must have been ≥ 2 x 10^6 CD34+ cells/kg


Biologic therapy

  • See Disease Characteristics


  • See Disease Characteristics
  • No chemotherapy after first transplantation

Endocrine therapy

  • Not specified


  • Not specified


  • Not specified



  • 18 to 65

Performance status

  • Southwest Oncology Group- 0-2

Life expectancy

  • More than 6 months


  • See Disease Characteristics


  • See Disease Characteristics


  • See Disease Characteristics


  • See Disease Characteristics
  • Left ventricular ejection fraction ≥ 45% by multiple gated acquisition scan or echocardiogram


  • diffusing capacity of lung for carbon monoxide ≥ 50%

Exclusion Criteria:

  • No myelodysplastic syndromes
  • No abnormal bone marrow cytogenetics


  • Not pregnant or nursing
  • Fertile patients must use effective contraception
  • Acceptable toxicity from first transplantation, confirmed by the transplant team
  • HIV negative
  • No other concurrent malignancy except treated skin cancer

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 identifier (NCT number): NCT00075608

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United States, Massachusetts
Boston University Cancer Research Center
Boston, Massachusetts, United States, 02118
Sponsors and Collaborators
Boston Medical Center
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Principal Investigator: Karen Quillen, MD Boston Medical Center

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Responsible Party: Karen Quillen, Medical Director, Blood Bank, Boston Medical Center Identifier: NCT00075608     History of Changes
Other Study ID Numbers: CDR0000347379
H-22603 ( Other Identifier: Boston University Medical Center IRB )
First Posted: January 12, 2004    Key Record Dates
Results First Posted: January 27, 2017
Last Update Posted: January 27, 2017
Last Verified: December 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Karen Quillen, Boston Medical Center:
primary systemic amyloidosis
Additional relevant MeSH terms:
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Immunosuppressive Agents
Multiple Myeloma
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Proteostasis Deficiencies
Metabolic Diseases
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Immunologic Factors
Physiological Effects of Drugs
Adjuvants, Immunologic