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Inhaled Sargramostim in Treating Patients With Melanoma Metastatic to the Lung

This study has been completed.
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: June 6, 2001
Last updated: November 29, 2012
Last verified: April 2007

June 6, 2001
November 29, 2012
May 2002
November 2012   (final data collection date for primary outcome measure)
  • Extent of up-regulation of specific anti-melanoma T cell frequencies (part A) [ Designated as safety issue: No ]
  • Maximum tolerated dose (part B) [ Designated as safety issue: Yes ]
Not Provided
Complete list of historical versions of study NCT00017121 on Archive Site
  • Toxicity profile [ Designated as safety issue: Yes ]
  • Progression-free survival [ Designated as safety issue: No ]
  • Overall survival [ Designated as safety issue: No ]
  • Objective response rate [ Designated as safety issue: No ]
  • Percent change from pretreatment levels in other immune parameters [ Designated as safety issue: No ]
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Inhaled Sargramostim in Treating Patients With Melanoma Metastatic to the Lung
Dose Finding Study of Aerosolized GM-CSF in the Treatment of Metastatic Melanoma to the Lung

RATIONALE: Inhaling sargramostim may interfere with the growth of tumor cells and may be an effective treatment for melanoma that has spread to the lung.

PURPOSE: This phase I trial is studying the side effects and best dose of inhaled sargramostim in treating patients with melanoma that is metastatic to the lung.


  • Determine immunomodulatory effects of aerosolized sargramostim (GM-CSF) in patients with metastatic melanoma to the lung (part A).
  • Determine toxicity profile of this therapy, in terms of pulmonary and hematologic toxicity, in these patients.
  • Determine, preliminarily, the therapeutic effects of this therapy, in terms of progression-free survival, overall survival, and objective response rate, in these patients.
  • Determine the maximum tolerated dose of GM-CSF in these patients (part B).

OUTLINE: This is a dose-escalation, multicenter study.

Patients receive aerosolized sargramostim (GM-CSF) twice a day on days 1-7 and 15-21. Treatment repeats every 28 days for 2 courses. Patients with no disease progression after completion of course 2 may continue on treatment until disease progression. Patients are grouped to 1 of 2 dose-escalation regimens (part A vs B).

  • Part A: Cohorts of 5-10 patients receive escalating doses of GM-CSF until the optimal immunostimulatory dose (ISD) is determined. The optimal ISD is defined as the dose at which at least 7 of 10 patients experience immunostimulation. Once the optimal ISD is determined, 10 patients receive aerosolized GM-CSF at a dose halfway between the optimal ISD and the preceding dose. Dose escalation is discontinued if at least 2 of 5 or at least 4 of 10 patients on a particular dose level experience dose-limiting toxicity.
  • Part B: Cohorts of 3-6 patients receive escalating doses of GM-CSF until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity. At least 6 patients are treated at the MTD.

After completion of study therapy, patients are followed at 3 months, every 2 months for 1 year, and then every 3-4 months for 5 years.

PROJECTED ACCRUAL: A total of 85 patients will be accrued for this study.

Phase 1
Primary Purpose: Treatment
  • Melanoma (Skin)
  • Metastatic Cancer
Biological: sargramostim
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Not Provided
November 2012   (final data collection date for primary outcome measure)


  • Histologically confirmed metastatic melanoma to the lung for which no known standard therapy exists
  • At least 1 unidimensionally measurable lesion
  • HLA-A2 positive (part A patients only)
  • Previously treated CNS metastases allowed provided there is no evidence of disease progression within the past 3 months



  • 18 and over

Performance status:

  • ECOG 0-2

Life expectancy:

  • At least 12 weeks


  • Absolute neutrophil count at least 1,000/mm^3
  • Platelet count at least 75,000/mm^3
  • Hemoglobin at least 8.0 g/dL


  • Bilirubin no greater than 2 times upper limit of normal (ULN)
  • AST no greater than 3 times ULN


  • Creatinine no greater than 2.5 times ULN


  • No New York Heart Association class III or IV heart disease


  • No pulmonary disease requiring concurrent active therapy (e.g., supplemental oxygen or bronchodilator)
  • FEV_1 at least 65% of predicted and at least 1.5 L


  • No known immunodeficiency state
  • No known autoimmune disease
  • No uncontrolled infection


  • No active psychotic disorder requiring pharmacotherapy
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception


Biologic therapy:

  • More than 2 weeks since prior biologic therapy
  • More than 2 weeks since prior immunotherapy
  • More than 4 weeks since prior filgrastim (G-CSF) or sargramostim (GM-CSF)
  • No other concurrent biologic therapy or immunotherapy
  • No concurrent G-CSF
  • No concurrent GM-CSF other than study drug


  • More than 3 weeks since prior chemotherapy (6 weeks for nitrosoureas or mitomycin)
  • No concurrent chemotherapy

Endocrine therapy:

  • More than 2 weeks since prior corticosteroids
  • No concurrent glucocorticosteroids


  • More than 2 weeks since prior radiotherapy
  • No concurrent radiotherapy


  • Not specified


  • More than 7 days since prior parenteral antibiotics
  • No concurrent parenteral antibiotics
  • No concurrent immunosuppressive agents
18 Years and older
Contact information is only displayed when the study is recruiting subjects
United States
CDR0000068654, NCCTG-N0071
Not Provided
Not Provided
North Central Cancer Treatment Group
National Cancer Institute (NCI)
Study Chair: Svetomir Markovic, MD, PhD Mayo Clinic
Investigator: John H. Donohue, MD Mayo Clinic
Investigator: Ellison F. Kalda, MD, FACS Avera Cancer Institute
Investigator: Lawrence J. Burgart, MD Mayo Clinic
Investigator: Axel Grothey, MD Mayo Clinic
Investigator: Muhammed Hussain, MD Saskatchewan Cancer Agency
National Cancer Institute (NCI)
April 2007

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP