Trial Outcomes & Findings for 3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Non-Myeloablative Therapy in Patients With High-Risk Neuroblastoma (NCT NCT01183429)

NCT ID: NCT01183429

Last Updated: 2019-08-13

Results Overview

on relapse-free survival in patients in first complete or very good partial remission, but at high risk of relapse.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

39 participants

Primary outcome timeframe

2 years

Results posted on

2019-08-13

Participant Flow

Participant milestones

Participant milestones
Measure
3F8 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. Clinical results will be compared to those in the predecessor trials which used only the standard 3F8 dosage. Starting with A(8), patients no longer receive high dose 3F8 but receive only standard dose 3F8 (20mg/m2/day) for all cycles. 3F8 and 13-cis-retinoic acid: 3F8 is dosed at 80 mg/m2/day (cycles 1-2) or 20 mg/m2/day (cycles 3 and beyond) and infused iv over 30-90 minutes. 13-cis-retinoic acid is dosed at 160 mg/m2/day, divided into two doses, x14 days. If a dose is missed, it can be made up at the end of the cycle. It is not taken on same days as 3F8. \*High-dose 3F8 will be administered only for patients enrolled on protocol from A(0) to A(7). Starting with A(8), patients receive standard dose (20mg/m2/day) during cycles 1 and 2.
Overall Study
STARTED
39
Overall Study
COMPLETED
39
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Consolidation of First Remission After Non-Myeloablative Therapy in Patients With High-Risk Neuroblastoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
3F8 and 13-cis-retinoic Acid
n=39 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. Clinical results will be compared to those in the predecessor trials which used only the standard 3F8 dosage. Starting with A(8), patients no longer receive high dose 3F8 but receive only standard dose 3F8 (20mg/m2/day) for all cycles. 3F8 and 13-cis-retinoic acid: 3F8 is dosed at 80 mg/m2/day (cycles 1-2) or 20 mg/m2/day (cycles 3 and beyond) and infused iv over 30-90 minutes. 13-cis-retinoic acid is dosed at 160 mg/m2/day, divided into two doses, x14 days. If a dose is missed, it can be made up at the end of the cycle. It is not taken on same days as 3F8. \*High-dose 3F8 will be administered only for patients enrolled on protocol from A(0) to A(7). Starting with A(8), patients receive standard dose (20mg/m2/day) during cycles 1 and 2.
Age, Categorical
<=18 years
39 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
Sex: Female, Male
Female
17 Participants
n=5 Participants
Sex: Female, Male
Male
22 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
2 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
36 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
5 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
1 Participants
n=5 Participants
Race (NIH/OMB)
White
28 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
5 Participants
n=5 Participants
Region of Enrollment
United States
33 Participants
n=5 Participants
Region of Enrollment
Colombia
1 Participants
n=5 Participants
Region of Enrollment
Greece
1 Participants
n=5 Participants
Region of Enrollment
India
1 Participants
n=5 Participants
Region of Enrollment
Singapore
1 Participants
n=5 Participants
Region of Enrollment
Spain
2 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Population: No data were collected

on relapse-free survival in patients in first complete or very good partial remission, but at high risk of relapse.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Population: No data were collected

to test the hypothesis that the minimal residual disease content of bone marrow 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 years

Population: No data were 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 and 13-cis-retinoic Acid

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Brian Kushner, MD

Memorial Sloan Kettering Cancer Center

Phone: 212-639-6793

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place