Trial Outcomes & Findings for Naxitamab and GM-CSF in Combination With IT in Patients With High-Risk Neuroblastoma (NCT NCT04560166)
NCT ID: NCT04560166
Last Updated: 2024-03-18
Results Overview
The proportion of patients obtaining a centrally assessed complete response (CR) or partial response (PR) according to the International Neuroblastoma Response Criteria (INRC)
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
2 participants
Primary outcome timeframe
84 days
Results posted on
2024-03-18
Participant Flow
Participant milestones
| Measure |
Naxitamab and GM-CSF in Combination With Irinotecan and Temozolomide
A treatment cycle is 21 days. The patients will receive irinotecan 50 mg/m2/day IV and temozolomide 100 mg/m2/day orally (both on Days 1-5) in combination with naxitamab 2.25 mg/kg/day IV (Days 2, 4, 8 and 10) (total 9 mg/kg per cycle), and GM-CSF 250 ug/m2/day sc, (Days 6-10).
Patients will receive up to 18 IT cycles after enrollment. Naxitamab and GM-CSF will be given for at least 8 cycles.
Naxitamab and GM-CSF in combination with irinotecan and temozolomide: • Irinotecan, solution for infusion (20 mg/mL)
* Temozolomide, capsules (5 mg, 20 mg and 100 mg)
* The humanized immunoglobulin isotype G (IgG1) monoclonal antibody (mAb) naxitamab, solution for infusion (4 mg/mL)
* Sargramostim (GM-CSF), lyophilized 250 µg single use vial (250 µg/vial)
|
|---|---|
|
Overall Study
STARTED
|
2
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Naxitamab and GM-CSF in Combination With IT in Patients With High-Risk Neuroblastoma
Baseline characteristics by cohort
| Measure |
Naxitamab and GM-CSF in Combination With Irinotecan and Temozolomide
n=2 Participants
A treatment cycle is 21 days. The patients will receive irinotecan 50 mg/m2/day IV and temozolomide 100 mg/m2/day orally (both on Days 1-5) in combination with naxitamab 2.25 mg/kg/day IV (Days 2, 4, 8 and 10) (total 9 mg/kg per cycle), and GM-CSF 250 ug/m2/day sc, (Days 6-10).
Patients will receive up to 18 IT cycles after enrollment. Naxitamab and GM-CSF will be given for at least 8 cycles.
Naxitamab and GM-CSF in combination with irinotecan and temozolomide: • Irinotecan, solution for infusion (20 mg/mL)
* Temozolomide, capsules (5 mg, 20 mg and 100 mg)
* The humanized immunoglobulin isotype G (IgG1) monoclonal antibody (mAb) naxitamab, solution for infusion (4 mg/mL)
* Sargramostim (GM-CSF), lyophilized 250 µg single use vial (250 µg/vial)
|
|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
4.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 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
|
2 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
|
0 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
South Korea
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 84 daysPopulation: The trial was terminated prematurely and no results from primary or secondary efficacy outcomes are available because images and bone marrow pathology were never submitted for central response assessment.
The proportion of patients obtaining a centrally assessed complete response (CR) or partial response (PR) according to the International Neuroblastoma Response Criteria (INRC)
Outcome measures
Outcome data not reported
Adverse Events
Naxitamab and GM-CSF in Combination With Irinotecan and Temozolomide
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Naxitamab and GM-CSF in Combination With Irinotecan and Temozolomide
n=2 participants at risk
A treatment cycle is 21 days. The patients will receive irinotecan 50 mg/m2/day IV and temozolomide 100 mg/m2/day orally (both on Days 1-5) in combination with naxitamab 2.25 mg/kg/day IV (Days 2, 4, 8 and 10) (total 9 mg/kg per cycle), and GM-CSF 250 ug/m2/day sc, (Days 6-10).
Patients will receive up to 18 IT cycles after enrollment. Naxitamab and GM-CSF will be given for at least 8 cycles.
Naxitamab and GM-CSF in combination with irinotecan and temozolomide: • Irinotecan, solution for infusion (20 mg/mL)
* Temozolomide, capsules (5 mg, 20 mg and 100 mg)
* The humanized immunoglobulin isotype G (IgG1) monoclonal antibody (mAb) naxitamab, solution for infusion (4 mg/mL)
* Sargramostim (GM-CSF), lyophilized 250 µg single use vial (250 µg/vial)
|
|---|---|
|
Investigations
Alanine aminotransferase increased
|
50.0%
1/2 • Number of events 1 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Investigations
Aspartate aminotransferase increased
|
50.0%
1/2 • Number of events 1 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Investigations
Neutrophil count decreased
|
50.0%
1/2 • Number of events 3 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
Other adverse events
| Measure |
Naxitamab and GM-CSF in Combination With Irinotecan and Temozolomide
n=2 participants at risk
A treatment cycle is 21 days. The patients will receive irinotecan 50 mg/m2/day IV and temozolomide 100 mg/m2/day orally (both on Days 1-5) in combination with naxitamab 2.25 mg/kg/day IV (Days 2, 4, 8 and 10) (total 9 mg/kg per cycle), and GM-CSF 250 ug/m2/day sc, (Days 6-10).
Patients will receive up to 18 IT cycles after enrollment. Naxitamab and GM-CSF will be given for at least 8 cycles.
Naxitamab and GM-CSF in combination with irinotecan and temozolomide: • Irinotecan, solution for infusion (20 mg/mL)
* Temozolomide, capsules (5 mg, 20 mg and 100 mg)
* The humanized immunoglobulin isotype G (IgG1) monoclonal antibody (mAb) naxitamab, solution for infusion (4 mg/mL)
* Sargramostim (GM-CSF), lyophilized 250 µg single use vial (250 µg/vial)
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
100.0%
2/2 • Number of events 8 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Gastrointestinal disorders
Abdominal pain
|
50.0%
1/2 • Number of events 30 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Gastrointestinal disorders
Diarrhoea
|
50.0%
1/2 • Number of events 2 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Gastrointestinal disorders
Nausea
|
50.0%
1/2 • Number of events 1 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Gastrointestinal disorders
Vomiting
|
100.0%
2/2 • Number of events 4 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
General disorders
Fatigue
|
50.0%
1/2 • Number of events 1 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
General disorders
Localised oedema
|
50.0%
1/2 • Number of events 1 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
General disorders
Pain
|
50.0%
1/2 • Number of events 27 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
General disorders
Pyrexia
|
100.0%
2/2 • Number of events 7 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
General disorders
Swelling
|
50.0%
1/2 • Number of events 1 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Infections and infestations
COVID-19
|
100.0%
2/2 • Number of events 2 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Infections and infestations
Nasopharyngitis
|
50.0%
1/2 • Number of events 1 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Infections and infestations
Sinusitis
|
50.0%
1/2 • Number of events 1 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Infections and infestations
Tonsillitis
|
50.0%
1/2 • Number of events 1 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Investigations
Alanine aminotransferase increased
|
100.0%
2/2 • Number of events 2 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Investigations
Aspartate aminotransferase increased
|
100.0%
2/2 • Number of events 2 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Investigations
Neutrophil count decreased
|
100.0%
2/2 • Number of events 18 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Investigations
Platelet count decreased
|
50.0%
1/2 • Number of events 7 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
50.0%
1/2 • Number of events 3 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
50.0%
1/2 • Number of events 1 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
50.0%
1/2 • Number of events 2 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
50.0%
1/2 • Number of events 1 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Skin and subcutaneous tissue disorders
Rash
|
50.0%
1/2 • Number of events 4 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
100.0%
2/2 • Number of events 24 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
|
Vascular disorders
Hypotension
|
100.0%
2/2 • Number of events 19 • From day of first IMP administration until 42 days after last IMP administration, an average of 34 weeks.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place