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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00089258
Recruitment Status : Completed
First Posted : August 5, 2004
Last Update Posted : January 17, 2013
National Cancer Institute (NCI)
Information provided by:
Memorial Sloan Kettering Cancer Center

August 4, 2004
August 5, 2004
January 17, 2013
July 2004
November 2006   (Final data collection date for primary outcome measure)
Disease response as assessed by PT-PC at the end of 4 courses
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Complete list of historical versions of study NCT00089258 on Archive Site
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Biological Therapy in Treating Patients With Neuroblastoma That Has Not Responded to Previous Treatment
Phase II Study of Anti-GD2 3F8 Antibody and Biologic Response Modifiers for High-risk Neuroblastoma

RATIONALE: Monoclonal antibodies, such as monoclonal antibody 3F8, can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. Beta-glucan, isotretinoin, and sargramostim may increase the effectiveness of monoclonal antibody 3F8 by making tumor cells more sensitive to the monoclonal antibody. Combining different types of biological therapy may kill more tumor cells.

PURPOSE: This phase II trial is studying how well giving beta-glucan, isotretinoin, and sargramostim together with monoclonal antibody 3F8 works in treating patients with neuroblastoma that has not responded to previous treatment.


  • 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.
  • 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.
  • Determine the toxicity of this regimen in these patients.

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.

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

Phase 2
Masking: None (Open Label)
Primary Purpose: Treatment
  • Biological: beta-glucan
  • Biological: monoclonal antibody 3F8
  • Biological: sargramostim
  • Drug: isotretinoin
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
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November 2006   (Final data collection date for primary outcome measure)


  • 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



  • Any age

Performance status

  • Not specified

Life expectancy

  • Not specified


  • Not specified


  • No severe hepatic toxicity ≥ grade 3


  • No severe renal toxicity ≥ grade 3


  • No severe cardiac toxicity ≥ grade 3


  • No severe pulmonary toxicity ≥ grade 3


  • 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


Biologic therapy

  • Not specified


  • See Disease Characteristics

Endocrine therapy

  • Not specified


  • Not specified


  • Not specified
Sexes Eligible for Study: All
Child, Adult, Senior
Contact information is only displayed when the study is recruiting subjects
United States
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Memorial Sloan Kettering Cancer Center
National Cancer Institute (NCI)
Study Chair: Nai-Kong V. Cheung, MD, PhD Memorial Sloan Kettering Cancer Center
Memorial Sloan Kettering Cancer Center
January 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP