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3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Non-Myeloablative Therapy in Patients With High-Risk Neuroblastoma

Phase 2
Completed
Conditions
Neuroblastoma
Interventions
Registration Number
NCT01183429
Lead Sponsor
Memorial Sloan Kettering Cancer Center
Brief Summary

The purpose of this study is to find out what effects, good and/or bad, the combination of 3F8 and GM-CSF has on the patient and the cancer.

Antibodies are made by the body to attack tumors and to fight infections. 3F8 is the name of one kind of antibody. It is made by mice, and it can attack neuroblastoma in people. 3F8 has been used safely in many patients, and it has killed cancer cells in some patients. One way it can kill cancer cells is by causing the patient's own white blood cells to attack the cancer. Granulocytes are one kind of white blood cell. GM-CSF increases the number of granulocytes in people, and it makes the granulocytes better able to kill the cancer cells.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
39
Inclusion Criteria
  • Diagnosis of NB as defined by a) histopathology (confirmed by the MSKCC Department of Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine levels.
  • High-risk NB as defined by risk-related treatment guidelines1 and the International NB Staging System,89 i.e., stage 4 with (any age) or without (≥18 months of age) MYCN amplification, MYCN-amplified stage 2 or stage 3 (any age), or MYCN-amplified stage 4S.
  • The patients are in first CR/VGPR after conventional therapy. They have no measurable MIBG-avid soft tissue tumor assessable for response.
  • Signed informed consent indicating awareness of the investigational nature of this program.
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Exclusion Criteria
  • Creatinine > 3.0 mg/dL
  • ALT, AST and Alkaline Phosphatase > 5.0 times the upper limit of normal
  • Bilirubin > 3.0 mg/dL
  • Patients with grade 3 or higher toxicities (using the CTCAE v3.0) related to cardiac, neurological, pulmonary or gastrointestinal function as determined by physical exam. Patients must have normal blood pressure for age.
  • Progressive disease
  • History of allergy to mouse proteins.
  • Active life-threatening infection.
  • Human anti-mouse antibody (HAMA) titer >1000 Elisa units/ml.
  • Inability to comply with protocol requirements
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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
3F8 and 13-cis-retinoic acid3F8 and 13-cis-retinoic acidThis phase II, open-label, single arm trial assesses the anti-NB activity of high-dose 3F8 (80 mg/m2/day), which is used in cycles 1-2, with return to standard 3F8 dosage (20 mg/m2/day) in subsequent cycles. Clinical results will be compared to those in the predecessor trials which used only the standard 3F8 dosage. Starting with A(8), patients no longer receive high dose 3F8 but receive only standard dose 3F8 (20mg/m2/day) for all cycles.
Primary Outcome Measures
NameTimeMethod
Assess the Impact of High-dose 3F8/GM-CSF2 years

on relapse-free survival in patients in first complete or very good partial remission, but at high risk of relapse.

Secondary Outcome Measures
NameTimeMethod
Apply Real-time Quantitative RT-PCR2 years

to test the hypothesis that the minimal residual disease content of bone marrow after the first treatments with 3F8/GMCSF has significant prognostic impact on relapse-free survival.

Monitor Safety of the High-dose Antibody Treatment2 years

to assure no side-effects or noxious sequelae develop or emerge that were not seen in the prior phase I study.

Trial Locations

Locations (1)

Memorial Sloan Kettering Cancer Center

🇺🇸

New York, New York, United States

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