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A Phase II Study of Aerosolized GM-CSF (NSC# 613795, IND# 11042) in Patients With First Pulmonary Recurrence of Osteosarcoma

Phase 2
39 Years
Not Enrolling
Metastatic Cancer, Sarcoma

Thank you

Trial Information

A Phase II Study of Aerosolized GM-CSF (NSC# 613795, IND# 11042) in Patients With First Pulmonary Recurrence of Osteosarcoma



- Assess the histological findings from patients with first pulmonary recurrence of
osteosarcoma who undergo resection of pulmonary metastases after treatment with 2
courses of aerosolized sargramostim (GM-CSF).

- Determine the event-free survival of patients treated with this drug.

- Determine whether the maximum tolerated dose in the trial of inhaled GM-CSF in adult
patients with melanoma is tolerable in pediatric patients.


- Determine the effect of specific thoracic surgical management on outcome in patients
treated with this drug.

OUTLINE: This is a multicenter, dose escalation study. Patients are assigned to 1 of 2
groups according to the extent of pulmonary recurrence (unilateral or bilateral).

- Group I (unilateral recurrence):

- Initial inhalation therapy: Patients receive inhaled sargramostim (GM-CSF) twice
daily on days 1-7. Treatment repeats every other week every 14 days for a total of
2 courses.

- Thoracotomy: Patients undergo thoracotomy on day 22.

- Post-thoracotomy inhalation therapy: Beginning on day 29, or as soon as possible
thereafter, patients resume inhalation therapy as above for up to 12 additional

- Group II (bilateral recurrence): Patients may be enrolled on study either before or
after the first thoracotomy.

- First thoracotomy: Patients undergo unilateral thoracotomy.

- Initial inhalation therapy: Patients receive inhaled GM-CSF, as soon as possible
after recovery from first thoracotomy, twice daily on days 1-7. Treatment repeats
every other week every 14 days for a total of 2 courses.

- Contralateral thoracotomy: Patients undergo contralateral thoracotomy on day 22.

- Post-thoracotomy inhalation therapy: Beginning on day 29, or as soon as possible,
patients resume inhalation therapy as above for up to 12 additional courses.

Treatment in both groups continues in the absence of disease progression or unacceptable

Patients are followed every 2 months for 1 year, every 4 months for 1 year, every 6 months
for 3 years, and then annually thereafter.

PROJECTED ACCRUAL: A total of 40 patients will be accrued for this study within 1.6-2 years.

Inclusion Criteria


- Histologically confirmed osteosarcoma at primary diagnosis

- Lesions detected in at least 1 lung that are consistent with metastatic disease
and approachable with thoracotomy

- No prior recurrence of osteosarcoma

- No other sites of metastases

- Resectable pulmonary nodule(s), defined as nodule(s) that are removable without
performing a pneumonectomy (e.g., nodules immediately adjacent to the main stem
bronchus or main pulmonary vessels)

- Prior thoracotomy allowed in patients with imaging consistent with metastatic
involvement in both lungs provided the lung on which the thoracotomy was performed is

- No pleural effusion or pleural based nodules



- 39 and under

Performance status

- Karnofsky 50-100% (patients over 16 years of age)

- Lansky 50-100% (patients 16 years of age and under)

Life expectancy

- At least 8 weeks


- Not specified


- Not specified


- Not specified


- No evidence of dyspnea at rest

- No exercise intolerance

- Pulse oximetry at least 94%

- Baseline FEV_1 at least 80% of predicted

- No history of asthma

- No history of reactive airway disease

- No history of bronchospasm


- Willing and able to perform inhalation therapy

- No medical contraindication to surgical excision

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception


Biologic therapy

- No other concurrent immunotherapy

- No other concurrent immunomodulating agents


- No concurrent anticancer chemotherapy

Endocrine therapy

- No concurrent steroids by any route


- Not specified


- See Disease Characteristics

- No concurrent thoracoscopy or video-assisted thoracic surgery


- No more than 1 prior treatment regimen for osteosarcoma

- No concurrent participation in another COG therapeutic study

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Expression of Fas/Fas ligand, presence of dendritic cells, and macrophage infiltration by immunohistochemical analysis before and after the inhalation therapy that follows thoracotomy

Outcome Time Frame:

Up to 6 years

Safety Issue:


Principal Investigator

Carola A. S. Arndt, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Mayo Clinic


United States: Federal Government

Study ID:




Start Date:

July 2004

Completion Date:

Related Keywords:

  • Metastatic Cancer
  • Sarcoma
  • metastatic osteosarcoma
  • recurrent osteosarcoma
  • lung metastases
  • Neoplasm Metastasis
  • Neoplasms
  • Neoplasms, Second Primary
  • Osteosarcoma
  • Recurrence
  • Sarcoma



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Barbara Ann Karmanos Cancer Institute Detroit, Michigan  48201
Duke Comprehensive Cancer Center Durham, North Carolina  27710
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University of Texas Health Science Center at San Antonio San Antonio, Texas  78284-7811
Van Elslander Cancer Center at St. John Hospital and Medical Center Grosse Pointe Woods, Michigan  48236
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University of Wisconsin Paul P. Carbone Comprehensive Cancer Center Madison, Wisconsin  53792-6164
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Newark Beth Israel Medical Center Newark, New Jersey  07112
CCOP - Scott and White Hospital Temple, Texas  76508
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Baltimore, Maryland  21231-2410
Cleveland Clinic Taussig Cancer Center Cleveland, Ohio  44195
Children's National Medical Center Washington, District of Columbia  20010-2970
Children's Mercy Hospital Kansas City, Missouri  64108
Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania  15213
Children's Hospital and Regional Medical Center - Seattle Seattle, Washington  98105
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Miami Children's Hospital Miami, Florida  33155-4069
All Children's Hospital St. Petersburg, Florida  33701
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Palmetto Health South Carolina Cancer Center Columbia, South Carolina  29203
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Covenant Children's Hospital Lubbock, Texas  79410
Midwest Children's Cancer Center at Children's Hospital of Wisconsin Milwaukee, Wisconsin  53226
Arizona Cancer Center at University of Arizona Health Sciences Center Tucson, Arizona  85724
Arkansas Cancer Research Center at University of Arkansas for Medical Sciences Little Rock, Arkansas  72205
Kansas Masonic Cancer Research Institute at the University of Kansas Medical Center Kansas City, Kansas  66160-7353
Blumenthal Cancer Center at Carolinas Medical Center Charlotte, North Carolina  28232-2861
Loma Linda University Cancer Institute at Loma Linda University Medical Center Loma Linda, California  92354
Jonathan Jaques Children's Cancer Center at Miller Children's Hospital Long Beach, California  90801
Lee Cancer Care of Lee Memorial Health System Fort Myers, Florida  33901
University of Florida Shands Cancer Center Gainesville, Florida  32610-0232
Baptist-South Miami Regional Cancer Program Miami, Florida  33176
Nemours Children's Clinic - Orlando Orlando, Florida  32806
Sacred Heart Cancer Center at Sacred Heart Hospital Pensacola, Florida  32504
St. Joseph's Cancer Institute at St. Joseph's Hospital Tampa, Florida  33607
Kaplan Cancer Center at St. Mary's Medical Center West Palm Beach, Florida  33407
Winship Cancer Institute of Emory University Atlanta, Georgia  30322
Alvin and Lois Lapidus Cancer Institute at Sinai Hospital Baltimore, Maryland  21215
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Providence Cancer Center at Sacred Heart Medical Center Spokane, Washington  99220-2555
St. Vincent Hospital Regional Cancer Center Green Bay, Wisconsin  54307-3508
James P. Wilmot Cancer Center at University of Rochester Medical Center Rochester, New York  14642
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UCSF Helen Diller Family Comprehensive Cancer Center San Francisco, California  94115
Alfred I. duPont Hospital for Children Wilmington, Delaware  19803
Lombardi Comprehensive Cancer Center at Georgetown University Medical Center Washington, District of Columbia  20007
Curtis and Elizabeth Anderson Cancer Institute at Memorial Health University Medical Center Savannah, Georgia  31403-3089
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Simmons Cooper Cancer Institute Springfield, Illinois  62794-9677
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Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center New York, New York  10032
Akron Children's Hospital Akron, Ohio  44308-1062
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Oklahoma University Cancer Institute Oklahoma City, Oklahoma  73104
Legacy Emanuel Hospital and Health Center and Children's Hospital Portland, Oregon  97227
M. D. Anderson Cancer Center at University of Texas Houston, Texas  77030-4009
Stanford Cancer Center Stanford, California  94305-5824
University of Miami Sylvester Comprehensive Cancer Center - Miami Miami, Florida  33136
Lehigh Valley Hospital - Muhlenberg Bethlehem, Pennsylvania  18017
Lurleen Wallace Comprehensive Cancer at University of Alabama - Birmingham Birmingham, Alabama  35294
Greenville Hospital Cancer Center Greenville, South Carolina  29605
Oregon Health and Science University Cancer Institute Portland, Oregon  97239-3098