Back to resultsA Study of the Effect of Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin in Patients in First Remission of High-Risk Neuroblastoma
Primary Purpose
Neuroblastoma
Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Hu3F8
GM-CSF
Isotretinoin
Sponsored by
About this trial
This is an interventional treatment trial for Neuroblastoma focused on measuring Hu3F8, GM-CSF, Isotretinoin, 16-1643
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of NB as defined by a) histopathology (confirmed by the MSK Department of Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine levels.
- Patients must have high-risk NB (MYCN-amplified stage 2/3/4/4S of any age and MYCN-nonamplified stage 4 in patients greater than 18 months of age).
- Patients must be in first CR/VGPR
- Patients must have a negative human anti-hu3F8 antibody (HAHA) titer
Exclusion Criteria:
- Existing major organ dysfunction, i.e., renal., cardiac, hepatic, neurologic, pulmonary, or gastrointestinal toxicity ≥ grade 3, with the exception of hearing loss and hematologic toxicity.
- Active life-threatening infection.
- Inability to comply with protocol requirements
Sites / Locations
- Memorial Sloan Kettering Cancer Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Hu3F8/GM-CSF Plus Isotretinoin
Arm Description
In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF x5 cycles and isotretinoin x6 cycles. Isotretinoin starts after cycle 2 of hu3F8/GM-CSF.
Outcomes
Primary Outcome Measures
relapse-free survival (RFS)
Patients are considered a response failure under this protocol if progressive disease is evident before two years. Deaths from toxicity attributable to protocol treatment will be counted as events. Patients who withdraw or are lost to followup before two years will be considered as progressions.
Secondary Outcome Measures
Full Information
NCT ID
NCT03033303
First Posted
January 24, 2017
Last Updated
July 3, 2023
Sponsor
Memorial Sloan Kettering Cancer Center
Collaborators
Y-mAbs Therapeutics
1. Study Identification
Unique Protocol Identification Number
NCT03033303
Brief Title
A Study of the Effect of Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin in Patients in First Remission of High-Risk Neuroblastoma
Official Title
Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin for Consolidation of First Remission of Patients With High-Risk Neuroblastoma: A Phase II Study
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
January 23, 2017 (Actual)
Primary Completion Date
June 2024 (Anticipated)
Study Completion Date
June 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Memorial Sloan Kettering Cancer Center
Collaborators
Y-mAbs Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
The purpose of this study is to test see the combined effects of the study drug called Humanized 3F8 (Hu3F8) when used with granulocyte-macrophage colony stimulating factor (GM-CSF). Hu3F8 plus GM-CSF could prevent your neuroblastoma from growing, but it could also cause side effects.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuroblastoma
Keywords
Hu3F8, GM-CSF, Isotretinoin, 16-1643
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF.
Masking
None (Open Label)
Allocation
N/A
Enrollment
59 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Hu3F8/GM-CSF Plus Isotretinoin
Arm Type
Experimental
Arm Description
In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF x5 cycles and isotretinoin x6 cycles. Isotretinoin starts after cycle 2 of hu3F8/GM-CSF.
Intervention Type
Biological
Intervention Name(s)
Hu3F8
Intervention Description
Day 1: hu3F8 infused iv over ~30 to 90 minutes. Day 3: hu3F8 infused iv over ~30 to 90 minutes. Day 5: hu3F8 infused iv over ~30 to 90 minutes.
Intervention Type
Drug
Intervention Name(s)
GM-CSF
Intervention Description
Days -4 to 0: GM-CSF 250 mcg/m2/day, subcutaneously. Days 1 to 5: GM-CSF 500 mcg/m2/day, subcutaneously.
Intervention Type
Drug
Intervention Name(s)
Isotretinoin
Intervention Description
Isotretinoin is administered at 160 mg/m2/d, divided into two doses, x14 days.
Primary Outcome Measure Information:
Title
relapse-free survival (RFS)
Description
Patients are considered a response failure under this protocol if progressive disease is evident before two years. Deaths from toxicity attributable to protocol treatment will be counted as events. Patients who withdraw or are lost to followup before two years will be considered as progressions.
Time Frame
2 years
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of NB as defined by a) histopathology (confirmed by the MSK Department of Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine levels.
Patients must have high-risk NB (MYCN-amplified stage 2/3/4/4S of any age and MYCN-nonamplified stage 4 in patients greater than 18 months of age).
Patients must be in first CR/VGPR
Patients must have a negative human anti-hu3F8 antibody (HAHA) titer
Exclusion Criteria:
Existing major organ dysfunction, i.e., renal., cardiac, hepatic, neurologic, pulmonary, or gastrointestinal toxicity ≥ grade 3, with the exception of hearing loss and hematologic toxicity.
Active life-threatening infection.
Inability to comply with protocol requirements
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Brian H. Kushner, MD
Organizational Affiliation
Memorial Sloan Kettering Cancer Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Memorial Sloan Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
12. IPD Sharing Statement
Links:
URL
http://www.mskcc.org/mskcc/html/44.cfm
Description
Memorial Sloan Kettering Cancer Center
Learn more about this trial
A Study of the Effect of Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin in Patients in First Remission of High-Risk Neuroblastoma
We'll reach out to this number within 24 hrs