Phase II Study of Anti-GD2 3F8 Antibody and GM-CSF for High-Risk Neuroblastoma
- Determine the efficacy of sargramostim (GM-CSF) in enhancing monoclonal antibody
3F8-mediated ablation in patients with high-risk neuroblastoma.
- Determine the prognostic impact of minimal residual bone marrow disease on relapse-free
survival of patients treated with this regimen.
- Compare the effects of short-term (2-hour intravenous) vs prolonged (subcutaneous
release) daily GM-CSF on granulocyte activation, in order to establish the optimal
route for tumor-cell kill in these patients.
OUTLINE: This is an open-label study. Patients are stratified according to evaluable disease
(yes [primary refractory bone marrow disease] vs no [no evidence of disease]).
Patients receive sargramostim (GM-CSF) subcutaneously on days -5 to 4 and monoclonal
antibody 3F8 IV over 0.5-1.5 hours on days 0-4. Treatment repeats every 3 weeks for 4
courses and then every 8 weeks for up to a total of 24 months in the absence of disease
progression or unacceptable toxicity.
Beginning after 2 courses of GM-CSF and monoclonal antibody 3F8, patients also receive oral
isotretinoin twice daily on days 1-14 (when no monoclonal antibody 3F8 is administered).
Treatment with isotretinoin repeats approximately every 28 days for 6 courses.
PROJECTED ACCRUAL: A total of 340 patients will be accrued for this study.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Efficacy at completion of treatment
Brian H. Kushner, MD
Memorial Sloan-Kettering Cancer Center
United States: Food and Drug Administration
|Memorial Sloan-Kettering Cancer Center||New York, New York 10021|