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

Alternate Title

Biological Therapy in Treating Patients With Neuroblastoma That Has Not Responded to Previous Treatment

Basic Trial Information

Phase	Type	Status	Age	Sponsor	Protocol IDs
Phase II	Treatment	Completed	Any age	NCI	MSKCC-04050 NCT00089258

Objectives

1. Determine the efficacy of beta-glucan, isotretinoin, and sargramostim (GM-CSF) in enhancing monoclonal antibody 3F8-mediated ablation in patients with high-risk refractory neuroblastoma.
2. Determine the antitumor activity of this regimen, in terms of assessing disease status in the bone marrow by real-time quantitative reverse transcription polymerase chain reaction, in these patients.
3. Determine the toxicity of this regimen in these patients.

Entry Criteria

Disease Characteristics:

• Diagnosis of neuroblastoma, as defined by 1 of the following:
  • Histologically confirmed disease
  • Bone marrow metastases plus high urine catecholamines



• High-risk disease meeting 1 of the following stage criteria:
  • Stage IV, with 1 of the following:
    • Any age with MYCN amplification
    • > 18 months of age without MYCN amplification
  • Stage III, with both of the following:
    • Any age with MYCN amplification
    • Unresectable disease
  • Stage 4S with MYCN amplification



• Measurable or evaluable soft tissue disease



• Relapsed disease resistant to standard induction chemotherapy and salvage therapy

Prior/Concurrent Therapy:

Biologic therapy

• Not specified

Chemotherapy

• See Disease Characteristics

Endocrine therapy

• Not specified

Radiotherapy

• Not specified

Surgery

• Not specified

Patient Characteristics:

Age

• Any age

Performance status

• Not specified

Life expectancy

• Not specified

Hematopoietic

• Not specified

Hepatic

• No severe hepatic toxicity ≥ grade 3

Renal

• No severe renal toxicity ≥ grade 3

Cardiovascular

• No severe cardiac toxicity ≥ grade 3

Pulmonary

• No severe pulmonary toxicity ≥ grade 3

Other

• Not pregnant
• Negative pregnancy test
• No severe neurologic toxicity ≥ grade 3
• No severe gastrointestinal toxicity ≥ grade 3
• No other severe major organ dysfunction except ototoxicity
• No history of allergy to mouse proteins
• No active life-threatening infection
• No human anti-mouse antibody titer > 1,000 ELISA units/mL

Expected Enrollment

A total of 27-74 patients (10-33 for stratum 1 and 17-41 for stratum 2) will be accrued for this study.

Outcomes

Disease response as assessed by PT-PC at the end of 4 courses

Outline

This is an open-label study. Patients are stratified according to refractory disease (primary refractory [never had disease progression or disease recurrence] vs secondary refractory [recurrent disease that did not respond completely to reinduction therapy]).

• Courses 1 and 2: Patients receive sargramostim (GM-CSF) subcutaneously once daily on days -5 to 11. Patients also receive oral beta-glucan once daily on days -2 to 11 and monoclonal antibody (MOAB) 3F8 IV over 30-90 minutes on days 0-4 and 7-11.



• Courses 3 and 4: Patients receive GM-CSF, beta-glucan, and MOAB 3F8 as above. Patients also receive oral isotretinoin twice daily on days -2 to 11.

Treatment repeats every 2-4 weeks for 4 courses in the absence of disease progression or unacceptable toxicity.

Patients are followed every 3 months for 2 years.

Trial Contact Information

Trial Lead Organizations

Memorial Sloan-Kettering Cancer Center

Nai-Kong Cheung, MD, PhD, Protocol chair		Ph: 212-639-8401; 800-525-2225

Registry Information
Official Title		Phase II Study of anti-GD2 3F8 antibody and biologic response modifiers for high-risk neuroblastoma
Trial Start Date		2004-07-02
Trial Completion Date		2006-11-28
Registered in ClinicalTrials.gov		NCT00089258
Date Submitted to PDQ		2004-07-02
Information Last Verified		2007-04-29
NCI Grant/Contract Number		CA08748

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