High-Dose 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Myeloablative Therapy and Autologous Stem-Cell Transplantation

Phase 2

Completed

• Conditions: Neuroblastoma
• Interventions: Drug: 3F8 monoclonal antibody and 13-cis-Retinoic Acid

• Registration Number: NCT01183416
• Lead Sponsor: Memorial Sloan Kettering Cancer Center

Brief Summary

The purpose of this study is to see if high-dose 3F8 combined with GM-CSF is better than standard dose 3F8 in treating neuroblastoma. Another purpose of the study is to find out what effects, good and/or bad, 3F8 has on cancer. The investigators also want to see if the antibody works against a very small amount of neuroblastoma (minimal residual disease) that is left in the bone marrow.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-08
• Study First Submitted: 2010-08-16
• Study First Submitted QC: 2010-08-16
• Study First Posted: 2010-08-17
• Status Verified: 2018-10
• Primary Completion: 2018-10-31
• Study Completion: 2018-10-31
• Results First Submitted: 2019-09-16
• Results First Submitted QC: 2019-09-16
• Last Update Submitted: 2019-09-16
• Results First Posted: 2019-10-09
• Last Update Posted: 2019-10-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 4

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, MYCN-amplified stage 2 or stage 3 (any age), or MYCN-amplified stage 4S.
• The patients are post-stem cell transplantation and in first CR/VGPR, including 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

Group	Intervention	Description
3F8 monoclonal antibody and 13-cis-Retinoic Acid	3F8 monoclonal antibody and 13-cis-Retinoic Acid	This 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. The patients are post-transplant and in 1st complete/very good partial remission (CR/VGPR),89 with no evidence of NB by standard studies, but are at high risk for relapse.

Primary Outcome Measures

Name	Time	Method
Assess the Impact of High-dose 3F8/GM-CSF on Relapse-free Survival	2 years	in patients who are post-stem-cell transplantation and in first complete or very good partial remission, but at high risk of relapse.

Secondary Outcome Measures

Name	Time	Method
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.
Apply Real-time Quantitative RT-PCR to Test the Hypothesis That the Minimal Residual Disease Content of Bone Marrow	2 years	after the first treatments with 3F8/GMCSF has significant prognostic impact on relapse-free survival.

Trial Locations

Locations (1)

Memorial Sloan-Kettering Cancer Center; 🇺🇸 New York, New York, United States