Trial Information

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

OBJECTIVES:

Primary

- 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.

Secondary

- 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
courses.

- 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
toxicity.

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.