3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Primary Refractory Neuroblastoma in Bone Marrow

Phase 2

Completed

• Conditions: Neuroblastoma
• Interventions: Biological: 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic

• Registration Number: NCT01183897
• Lead Sponsor: Memorial Sloan Kettering Cancer Center

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2010-08-12
• Study First Submitted: 2010-08-16
• Study First Submitted QC: 2010-08-17
• Study First Posted: 2010-08-18
• Status Verified: 2018-09
• Primary Completion: 2018-09-25
• Study Completion: 2018-09-25
• Results First Submitted: 2019-07-24
• Results First Submitted QC: 2019-07-24
• Last Update Submitted: 2019-07-24
• Results First Posted: 2019-08-13
• Last Update Posted: 2019-08-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 31

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 (> or = to 18 months of age) MYCN amplification or MYCN-amplified stage 4S.
• Patients have primary refractory disease limited to BM, i.e., high-risk NB (defined above) resistant to standard therapy, as evidenced by incomplete response in BM, but no measurable MIBG-avid soft tissue tumor assessable for response and no progressive disease.
• Signed informed consent indicating awareness of the investigational nature of this program.

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 v 4.0) 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic	3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic	This phase II study of the anti-GD2 murine IgG3 monoclonal antibody 3F8 combined with granulocyte-macrophage colony stimulating factor (GM-CSF) will assess response in of primary refractory neuroblastoma in bone marrow (i.e., incomplete response to standard treatment).

Primary Outcome Measures

Name	Time	Method
Assess the Activity of High-dose 3F8/GM-CSF	2 years	against persistent neuroblastoma in bone marrow of patients who have no other evidence of disease by standard studies.

Secondary Outcome Measures

Name	Time	Method
Apply Real-time Quantitative RT-PCR	2 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 progression-free survival.
Monitor Safety of the High-dose Antibody Treatment	2 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