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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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