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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Boston Medical Center
ClinicalTrials.gov Identifier:
NCT00075621
First received: January 9, 2004
Last updated: July 25, 2013
Last verified: July 2013
  Purpose

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 and Plasma Cell Neoplasm
Drug: filgrastim
Drug: melphalan
Procedure: autologous peripheral blood stem cell transplantation
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
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 2015
Primary Completion Date: June 2005 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
tandem transplant
All patients will have tandem transplants, unless Complete response is achieved after the first 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
Other Name: alkeran
Procedure: autologous peripheral blood stem cell transplantation
autologous peripheral blood stem cell transplantation

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.

  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, 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
  • 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)

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
  • 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

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
  • 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

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: 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

Additional Information:
No publications provided

Responsible Party: Boston Medical Center
ClinicalTrials.gov Identifier: NCT00075621     History of Changes
Other Study ID Numbers: CDR0000347381, BUMC-2000-0279
Study First Received: January 9, 2004
Last Updated: July 25, 2013
Health Authority: United States: Federal Government

Keywords provided by Boston Medical Center:
primary systemic amyloidosis

Additional relevant MeSH terms:
Amyloidosis
Neoplasms
Multiple Myeloma
Neoplasms, Plasma Cell
Plasmacytoma
Proteostasis Deficiencies
Metabolic Diseases
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Melphalan
Lenograstim
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 April 17, 2014