Trial Outcomes & Findings for High-Dose 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Myeloablative Therapy and Autologous Stem-Cell Transplantation (NCT NCT01183416)
NCT ID: NCT01183416
Last Updated: 2019-10-09
Results Overview
in patients who are post-stem-cell transplantation and in first complete or very good partial remission, but at high risk of relapse.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
4 participants
Primary outcome timeframe
2 years
Results posted on
2019-10-09
Participant Flow
Participant milestones
| Measure |
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.
3F8 monoclonal antibody and 13-cis-Retinoic Acid: A cycle consists of treatment with 3F8 for 5 days. GMCSF is started 5 days in advance of each 3F8 cycle. The break between end of a cycle of 3F8/GM-CSF and start of next cycle is 2-to-4-weeks through 4 cycles; subsequent breaks are \~6-8 weeks. 13-cis-retinoic acid is started after cycle 2.
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
High-Dose 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Myeloablative Therapy and Autologous Stem-Cell Transplantation
Baseline characteristics by cohort
| Measure |
3F8 Monoclonal Antibody and 13-cis-Retinoic Acid
n=4 Participants
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.
3F8 monoclonal antibody and 13-cis-Retinoic Acid: A cycle consists of treatment with 3F8 for 5 days. GMCSF is started 5 days in advance of each 3F8 cycle. The break between end of a cycle of 3F8/GM-CSF and start of next cycle is 2-to-4-weeks through 4 cycles; subsequent breaks are \~6-8 weeks. 13-cis-retinoic acid is started after cycle 2.
|
|---|---|
|
Age, Continuous
|
4 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 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
|
0 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
4 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsPopulation: Data were not collected
in patients who are post-stem-cell transplantation and in first complete or very good partial remission, but at high risk of relapse.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 yearsPopulation: Data were not collected
after the first treatments with 3F8/GMCSF has significant prognostic impact on relapse-free survival.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 yearsPopulation: Data were not collected
to assure no side-effects or noxious sequelae develop or emerge that were not seen in the prior phase I study.
Outcome measures
Outcome data not reported
Adverse Events
3F8 Monoclonal Antibody and 13-cis-Retinoic Acid
Serious events: 1 serious events
Other events: 4 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
3F8 Monoclonal Antibody and 13-cis-Retinoic Acid
n=4 participants at risk
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.
3F8 monoclonal antibody and 13-cis-Retinoic Acid: A cycle consists of treatment with 3F8 for 5 days. GMCSF is started 5 days in advance of each 3F8 cycle. The break between end of a cycle of 3F8/GM-CSF and start of next cycle is 2-to-4-weeks through 4 cycles; subsequent breaks are \~6-8 weeks. 13-cis-retinoic acid is started after cycle 2.
|
|---|---|
|
Immune system disorders
Allergic Reaction
|
25.0%
1/4 • 2 years
|
|
Vascular disorders
Hypertension
|
25.0%
1/4 • 2 years
|
|
Vascular disorders
Vacular disorders - Other
|
25.0%
1/4 • 2 years
|
Other adverse events
| Measure |
3F8 Monoclonal Antibody and 13-cis-Retinoic Acid
n=4 participants at risk
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.
3F8 monoclonal antibody and 13-cis-Retinoic Acid: A cycle consists of treatment with 3F8 for 5 days. GMCSF is started 5 days in advance of each 3F8 cycle. The break between end of a cycle of 3F8/GM-CSF and start of next cycle is 2-to-4-weeks through 4 cycles; subsequent breaks are \~6-8 weeks. 13-cis-retinoic acid is started after cycle 2.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
100.0%
4/4 • 2 years
|
|
General disorders
Fever
|
100.0%
4/4 • 2 years
|
|
General disorders
Pain
|
100.0%
4/4 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
100.0%
4/4 • 2 years
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
100.0%
4/4 • 2 years
|
|
Gastrointestinal disorders
Nausea
|
75.0%
3/4 • 2 years
|
|
Gastrointestinal disorders
Vomiting
|
75.0%
3/4 • 2 years
|
|
Metabolism and nutrition disorders
Anorexia
|
50.0%
2/4 • 2 years
|
|
General disorders
Edema face
|
50.0%
2/4 • 2 years
|
|
Vascular disorders
Flushing
|
50.0%
2/4 • 2 years
|
|
Nervous system disorders
Lethargy
|
50.0%
2/4 • 2 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
50.0%
2/4 • 2 years
|
|
Renal and urinary disorders
Urinary retention
|
50.0%
2/4 • 2 years
|
|
Immune system disorders
Allergic reaction
|
25.0%
1/4 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
25.0%
1/4 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
25.0%
1/4 • 2 years
|
|
Gastrointestinal disorders
Constipation
|
25.0%
1/4 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
25.0%
1/4 • 2 years
|
|
Gastrointestinal disorders
Diarrhea
|
25.0%
1/4 • 2 years
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
25.0%
1/4 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
25.0%
1/4 • 2 years
|
|
Nervous system disorders
Headache
|
25.0%
1/4 • 2 years
|
|
Vascular disorders
Hypertension
|
25.0%
1/4 • 2 years
|
|
Vascular disorders
Hypotension
|
25.0%
1/4 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
25.0%
1/4 • 2 years
|
|
General disorders
Localized edema
|
25.0%
1/4 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
25.0%
1/4 • 2 years
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
25.0%
1/4 • 2 years
|
|
Reproductive system and breast disorders
Penile pain
|
25.0%
1/4 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Resp, thoracic & mediastinal disorder Other
|
25.0%
1/4 • 2 years
|
|
Cardiac disorders
Sinus tachycardia
|
25.0%
1/4 • 2 years
|
|
Skin and subcutaneous tissue disorders
Skin & subcutaneous tissue disorders - Other
|
25.0%
1/4 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Stridor
|
25.0%
1/4 • 2 years
|
|
Reproductive system and breast disorders
Vaginal pain
|
25.0%
1/4 • 2 years
|
|
Cardiac disorders
Ventricular arrhythmia
|
25.0%
1/4 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
25.0%
1/4 • 2 years
|
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