Trial Outcomes & Findings for Anti-GD2 3F8 Monoclonal Antibody and GM-CSF for High-Risk Neuroblastoma (NCT NCT02100930)
NCT ID: NCT02100930
Last Updated: 2020-02-28
Results Overview
Disease status is defined by the International Neuroblastoma Response Criteria - Complete response/remission (CR): NED; Partial response/remission: \>50% decrease in all disease parameters, exceptbone scan unchanged or improved; no more than 1 positive bone marrow site; Stable disease: \<50% decrease in all tumor markers; Progressive disease (PD): new lesion, or \>25 % increase in any disease marker.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
69 participants
Primary outcome timeframe
2 years
Results posted on
2020-02-28
Participant Flow
Participant milestones
| Measure |
Neuroblastoma
This is a single-arm, open label, open access study to provide the anti-GD2 murine IgG3 MoAb 3F8 combined with granulocyte-macrophage colony stimulating factor (GM-CSF) to patients with high-risk neuroblastoma (NB). This immunotherapy has shown efficacy against minimal residual disease (MRD) in such patients.
Anti-GD2 3F8 Monoclonal Antibody
GM-CSF (granulocyte-macrophage colony-stimulating factor)
oral isotretinoin
|
|---|---|
|
Overall Study
STARTED
|
69
|
|
Overall Study
COMPLETED
|
69
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Anti-GD2 3F8 Monoclonal Antibody and GM-CSF for High-Risk Neuroblastoma
Baseline characteristics by cohort
| Measure |
Neuroblastoma
n=69 Participants
This is a single-arm, open label, open access study to provide the anti-GD2 murine IgG3 MoAb 3F8 combined with granulocyte-macrophage colony stimulating factor (GM-CSF) to patients with high-risk neuroblastoma (NB). This immunotherapy has shown efficacy against minimal residual disease (MRD) in such patients.
Anti-GD2 3F8 Monoclonal Antibody
GM-CSF (granulocyte-macrophage colony-stimulating factor)
oral isotretinoin
|
|---|---|
|
Age, Continuous
|
4 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
29 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
40 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
68 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
52 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
7 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
69 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsDisease status is defined by the International Neuroblastoma Response Criteria - Complete response/remission (CR): NED; Partial response/remission: \>50% decrease in all disease parameters, exceptbone scan unchanged or improved; no more than 1 positive bone marrow site; Stable disease: \<50% decrease in all tumor markers; Progressive disease (PD): new lesion, or \>25 % increase in any disease marker.
Outcome measures
| Measure |
Neuroblastoma
n=69 Participants
This is a single-arm, open label, open access study to provide the anti-GD2 murine IgG3 MoAb 3F8 combined with granulocyte-macrophage colony stimulating factor (GM-CSF) to patients with high-risk neuroblastoma (NB). This immunotherapy has shown efficacy against minimal residual disease (MRD) in such patients.
Anti-GD2 3F8 Monoclonal Antibody
GM-CSF (granulocyte-macrophage colony-stimulating factor)
oral isotretinoin
|
|---|---|
|
Therapeutic Response
No Evidence of Disease
|
40 Participants
|
|
Therapeutic Response
Complete Response
|
3 Participants
|
|
Therapeutic Response
Partial Response
|
1 Participants
|
|
Therapeutic Response
Stable Disease
|
6 Participants
|
|
Therapeutic Response
Progression of Disease
|
19 Participants
|
PRIMARY outcome
Timeframe: 2 yearsDisease status is defined by the International Neuroblastoma Response Criteria - Complete response/remission (CR): NED; Partial response/remission: \>50% decrease in all disease parameters, exceptbone scan unchanged or improved; no more than 1 positive bone marrow site; Stable disease: \<50% decrease in all tumor markers; Progressive disease (PD): new lesion, or \>25 % increase in any disease marker.
Outcome measures
| Measure |
Neuroblastoma
n=69 Participants
This is a single-arm, open label, open access study to provide the anti-GD2 murine IgG3 MoAb 3F8 combined with granulocyte-macrophage colony stimulating factor (GM-CSF) to patients with high-risk neuroblastoma (NB). This immunotherapy has shown efficacy against minimal residual disease (MRD) in such patients.
Anti-GD2 3F8 Monoclonal Antibody
GM-CSF (granulocyte-macrophage colony-stimulating factor)
oral isotretinoin
|
|---|---|
|
Complete Remission
Stable Disease
|
6 Participants
|
|
Complete Remission
Progression of Disease
|
19 Participants
|
|
Complete Remission
No Evidence of Disease
|
40 Participants
|
|
Complete Remission
Complete Response
|
3 Participants
|
|
Complete Remission
Partial Response
|
1 Participants
|
Adverse Events
Neuroblastoma
Serious events: 26 serious events
Other events: 69 other events
Deaths: 16 deaths
Serious adverse events
| Measure |
Neuroblastoma
n=69 participants at risk
This is a single-arm, open label, open access study to provide the anti-GD2 murine IgG3 MoAb 3F8 combined with granulocyte-macrophage colony stimulating factor (GM-CSF) to patients with high-risk neuroblastoma (NB). This immunotherapy has shown efficacy against minimal residual disease (MRD) in such patients.
Anti-GD2 3F8 Monoclonal Antibody
GM-CSF (granulocyte-macrophage colony-stimulating factor)
oral isotretinoin
|
|---|---|
|
Gastrointestinal disorders
Abdominal Pain
|
1.4%
1/69 • 2 year
|
|
Metabolism and nutrition disorders
Acidosis
|
1.4%
1/69 • 2 year
|
|
Immune system disorders
Anaphylaxis
|
1.4%
1/69 • 2 year
|
|
Cardiac disorders
Cardiac arrest
|
1.4%
1/69 • 2 year
|
|
Gastrointestinal disorders
Constipation
|
1.4%
1/69 • 2 year
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
1.4%
1/69 • 2 year
|
|
Metabolism and nutrition disorders
Dehydration
|
1.4%
1/69 • 2 year
|
|
Gastrointestinal disorders
Diarrhea
|
2.9%
2/69 • 2 year
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
2.9%
2/69 • 2 year
|
|
General disorders
Fever
|
7.2%
5/69 • 2 year
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
1.4%
1/69 • 2 year
|
|
Vascular disorders
Hypertension
|
14.5%
10/69 • 2 year
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
2.9%
2/69 • 2 year
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
1.4%
1/69 • 2 year
|
|
Metabolism and nutrition disorders
Hypokalemia
|
1.4%
1/69 • 2 year
|
|
Vascular disorders
Hypotension
|
4.3%
3/69 • 2 year
|
|
General disorders
Hypothermia
|
1.4%
1/69 • 2 year
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
4.3%
3/69 • 2 year
|
|
Infections and infestations
Infections and infestations - Other, specify
|
11.6%
8/69 • 2 year
|
|
Nervous system disorders
Intracranial hemorrhage
|
1.4%
1/69 • 2 year
|
|
Investigations
Neutrophil count decreased
|
1.4%
1/69 • 2 year
|
|
Investigations
Platelet count decreased
|
2.9%
2/69 • 2 year
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
1.4%
1/69 • 2 year
|
|
Respiratory, thoracic and mediastinal disorders
Resp, thoracic & mediastinal disorder Other, spec
|
2.9%
2/69 • 2 year
|
|
Infections and infestations
Skin infection
|
4.3%
3/69 • 2 year
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
1.4%
1/69 • 2 year
|
|
Respiratory, thoracic and mediastinal disorders
Stridor
|
1.4%
1/69 • 2 year
|
|
Infections and infestations
Upper respiratory infection
|
1.4%
1/69 • 2 year
|
|
Renal and urinary disorders
Urinary retention
|
1.4%
1/69 • 2 year
|
|
Infections and infestations
Urinary tract infection
|
1.4%
1/69 • 2 year
|
|
Gastrointestinal disorders
Vomiting
|
1.4%
1/69 • 2 year
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
2.9%
2/69 • 2 year
|
|
Investigations
White blood cell decreased
|
1.4%
1/69 • 2 year
|
|
Infections and infestations
Wound infection
|
1.4%
1/69 • 2 year
|
Other adverse events
| Measure |
Neuroblastoma
n=69 participants at risk
This is a single-arm, open label, open access study to provide the anti-GD2 murine IgG3 MoAb 3F8 combined with granulocyte-macrophage colony stimulating factor (GM-CSF) to patients with high-risk neuroblastoma (NB). This immunotherapy has shown efficacy against minimal residual disease (MRD) in such patients.
Anti-GD2 3F8 Monoclonal Antibody
GM-CSF (granulocyte-macrophage colony-stimulating factor)
oral isotretinoin
|
|---|---|
|
General disorders
Edema face
|
8.7%
6/69 • 2 year
|
|
General disorders
Pain
|
8.7%
6/69 • 2 year
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
8.7%
6/69 • 2 year
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
8.7%
6/69 • 2 year
|
|
Gastrointestinal disorders
Abdominal pain
|
7.2%
5/69 • 2 year
|
|
Vascular disorders
Flushing
|
7.2%
5/69 • 2 year
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.2%
5/69 • 2 year
|
|
Gastrointestinal disorders
Vomiting
|
7.2%
5/69 • 2 year
|
|
Psychiatric disorders
Agitation
|
5.8%
4/69 • 2 year
|
|
Vascular disorders
Hypotension
|
5.8%
4/69 • 2 year
|
|
Psychiatric disorders
Anxiety
|
4.3%
3/69 • 2 year
|
|
Vascular disorders
Hypertension
|
4.3%
3/69 • 2 year
|
|
General disorders
Localized edema
|
4.3%
3/69 • 2 year
|
|
Gastrointestinal disorders
Nausea
|
4.3%
3/69 • 2 year
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
4.3%
3/69 • 2 year
|
|
Metabolism and nutrition disorders
Anorexia
|
2.9%
2/69 • 2 year
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.9%
2/69 • 2 year
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
2.9%
2/69 • 2 year
|
|
General disorders
Fever
|
2.9%
2/69 • 2 year
|
|
Nervous system disorders
Headache
|
2.9%
2/69 • 2 year
|
|
Psychiatric disorders
Irritability
|
2.9%
2/69 • 2 year
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
2.9%
2/69 • 2 year
|
|
Gastrointestinal disorders
Oral pain
|
2.9%
2/69 • 2 year
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
2.9%
2/69 • 2 year
|
Additional Information
Dr. Brian Kushner, MD
Memorial Sloan Kettering Cancer Center
Phone: 212-639-6793
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place