Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Second Autologous Stem Cell Transplant in Treating Patients With Persistent or Recurrent Primary Systemic (AL) Amyloidosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Boston Medical Center
ClinicalTrials.gov Identifier:
NCT00075608
First received: January 9, 2004
Last updated: December 9, 2011
Last verified: December 2011
  Purpose

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 Intervention Phase
Multiple Myeloma and Plasma Cell Neoplasm
Biological: filgrastim
Drug: melphalan
Procedure: autologous bone marrow transplantation
Procedure: peripheral blood stem cell transplantation
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase II Trial of Second Autologous Transplantation in AL Amyloidosis

Resource links provided by NLM:


Further study details as provided by Boston Medical Center:

Primary Outcome Measures:
  • Feasibility and tolerability [ Time Frame: 3 months after treatment and annually ] [ Designated as safety issue: Yes ]
  • Response and durability of response [ Time Frame: 3 months after treatment and annually ] [ Designated as safety issue: No ]
  • Evaluate immune reconstitution [ Time Frame: 3 months after treatment and annually ] [ Designated as safety issue: No ]

Enrollment: 12
Study Start Date: August 2001
Study Completion Date: October 2011
Primary Completion Date: October 2011 (Final data collection date for primary outcome measure)
Intervention Details:
    Biological: filgrastim
    16mcg/kg IV daily beginning three days prior to SCC through last day of SCC
    Other Name: G-CSF
    Drug: melphalan
    140-200 mcg/kg IV over two days
    Other Name: alkeran
    Procedure: autologous bone marrow transplantation
    infusion of previously collected stem cells on Day 0
    Procedure: peripheral blood stem cell transplantation
    infusion of previously collected stem cells on Day 0
Detailed Description:

OBJECTIVES:

  • 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.

OUTLINE:

  • 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.

  Eligibility

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

DISEASE CHARACTERISTICS:

  • 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
  • No myelodysplastic syndromes
  • No abnormal bone marrow cytogenetics
  • Prior stem cell yield must have been ≥ 2 x 10^6 CD34+ cells/kg

PATIENT CHARACTERISTICS:

Age

  • 18 to 65

Performance status

  • SWOG 0-2

Life expectancy

  • More than 6 months

Hematopoietic

  • See Disease Characteristics

Hepatic

  • See Disease Characteristics

Renal

  • See Disease Characteristics

Cardiovascular

  • See Disease Characteristics
  • LVEF ≥ 45% by MUGA or echocardiogram

Pulmonary

  • DLCO ≥ 50%

Other

  • 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

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • See Disease Characteristics

Chemotherapy

  • See Disease Characteristics
  • No chemotherapy after first transplantation

Endocrine therapy

  • Not specified

Radiotherapy

  • Not specified

Surgery

  • Not specified
  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: NCT00075608

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

Additional Information:
No publications provided

Responsible Party: Boston Medical Center
ClinicalTrials.gov Identifier: NCT00075608     History of Changes
Other Study ID Numbers: CDR0000347379, BUMC-2001-0156
Study First Received: January 9, 2004
Last Updated: December 9, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Boston Medical Center:
primary systemic amyloidosis

Additional relevant MeSH terms:
Amyloidosis
Multiple Myeloma
Neoplasms, Plasma Cell
Plasmacytoma
Blood Protein Disorders
Cardiovascular Diseases
Hematologic Diseases
Hemorrhagic Disorders
Hemostatic Disorders
Immune System Diseases
Immunoproliferative Disorders
Lymphoproliferative Disorders
Metabolic Diseases
Neoplasms
Neoplasms by Histologic Type
Paraproteinemias
Proteostasis Deficiencies
Vascular Diseases

ClinicalTrials.gov processed this record on November 19, 2014