Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Trial Contact Information
Registry Information

Alternate Title

Monoclonal Antibody 3F8 and Sargramostim in Treating Patients With Neuroblastoma

Basic Trial Information

Phase	Type	Status	Age	Sponsor	Protocol IDs
Phase II	Treatment	Active	Any age	NCI	MSKCC-03077 NCT00072358

Objectives

1. Determine the efficacy of sargramostim (GM-CSF) in enhancing monoclonal antibody 3F8-mediated ablation in patients with high-risk neuroblastoma.
2. Determine the prognostic impact of minimal residual bone marrow disease on relapse-free survival of patients treated with this regimen.
3. 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.

Entry Criteria

Disease Characteristics:

• 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

Prior/Concurrent Therapy:

• Not specified

Patient Characteristics:

• 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

Expected Enrollment

A total of 270 patients will be accrued for this study.

Outcomes

Efficacy at completion of treatment
Relapse-free survival every 3 months

Compare granulocyte activation in patients treated with short-term vs prolonged daily exposure to sargramostim (GM-CSF) after 4 courses
Simplify treatment with consequent reduction in cost

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.

Trial Contact Information

Trial Lead Organizations

Memorial Sloan-Kettering Cancer Center

Brian Kushner, MD, Principal investigator		Ph: 212-639-6793; 800-525-2225 Email: kushnerb@mskcc.org

U.S.A.
New York
	New York
	Memorial Sloan-Kettering Cancer Center
		Brian Kushner, MD	Ph:  212-639-6793 800-525-2225
			Email: kushnerb@mskcc.org
		Nai-Kong Cheung, MD, PhD	Ph:  212-639-8401 800-525-2225

Registry Information
Official Title		Phase II Study of Anti-GD2 3F8 Antibody and GM-CSF for High-Risk Neuroblastoma
Trial Start Date		2003-07-08
Trial Completion Date		2010-12-31 (estimated)
Registered in ClinicalTrials.gov		NCT00072358
Date Submitted to PDQ		2003-09-30
Information Last Verified		2009-09-14
NCI Grant/Contract Number		CA08748, CA105091

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