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Phase II Study of Anti-GD2 3F8 Antibody and GM-CSF for High-Risk Neuroblastoma

Phase 2
Open (Enrolling)

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Trial Information

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.

Inclusion Criteria


- Diagnosis of neuroblastoma by histopathology OR bone marrow metastases and high urine
catecholamine levels

- Disease must meet risk-related treatment guidelines and any of the following
International Neuroblastoma Staging System stages:

- Stage 4 with (any age) OR without (> 18 months of age of age) MYCN amplification

- MYCN-amplified other than stage 1

- No evidence of disease (i.e., in complete response/remission or very good partial
response/remission) OR disease resistant to standard therapy (i.e., incomplete
response in bone marrow)

- No progressive disease or MIBG-avid soft tissue tumor


- No existing renal, cardiac, hepatic, neurologic, pulmonary, or gastrointestinal
toxicity ≥ grade 3

- No human anti-mouse antibody (HAMA) titer greater than 1,000 Elisa units/mL

- No history of allergy to mouse proteins

- No active life-threatening infection

- Not pregnant

- Negative pregnancy test


- Not specified

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:

Efficacy at completion of treatment

Outcome Time Frame:

3 years

Safety Issue:


Principal Investigator

Brian H. Kushner, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Memorial Sloan-Kettering Cancer Center


United States: Food and Drug Administration

Study ID:




Start Date:

July 2003

Completion Date:

December 2013

Related Keywords:

  • Neuroblastoma
  • disseminated neuroblastoma
  • localized unresectable neuroblastoma
  • recurrent neuroblastoma
  • regional neuroblastoma
  • stage 4S neuroblastoma
  • Neuroblastoma



Memorial Sloan-Kettering Cancer Center New York, New York  10021