NCT02395666 · STUDY IN CONTEXT

Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission

Draft scope assessment: narrower. Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.

Read the full study record and all posted results →

Linked outcomes

Measures are not ranked. Open each original source section for treatment groups, denominators, units and results.

primary · POSTED

To evaluate the preventative activity of DFMO as a single agent in patients that are in remission based on: Event free survival (EFS)

One subject removed from Stratum 1 due to not fitting study criteria upon review

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secondary · POSTED

To evaluate the preventative activity of DFMO as a single agent in patients with neuroblastoma who are in remission based on: Overall Survival (OS)

Source null

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secondary · POSTED

To continue to determine the safety and tolerability of DFMO as a single agent and in pediatric and young adult patients with high risk neuroblastoma that is in remission.

Stratum 1 and 2 were analyzed together as one safety group as per statistical analysis plan.

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secondary · POSTED

Tests (p-value) of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype. Blood: microRNA analysis as predictor of DFMO effect, ornithine decarboxylase (ODC) single nucleotide polymorphism (SNP) analysis in DNA isolated from nucleated cells

Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.

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secondary · POSTED

Pharmacokinetic assay Cmax/D Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days.

12 subjects from both Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.

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secondary · POSTED

Pharmacokinetic assay AUC(0-6 hr)/D Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days

12 subjects from both Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.

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Reported safety

Serious events are not the same as severe events. Compare the original safety groups and their at-risk populations.

Open the study safety table →

Quality of life and patient-reported measures

No linked quality-of-life measure is recorded for this setting and study.

Full draft applicability and source-link record

trial

release id: clinical-indications-draft-1
disease state id: ds-neuroblastoma
nct id: NCT02395666
trial label: Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission
applicability: narrower
applicability note: Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded.
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z
provenance class: regulator-cited
discovery note: Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
outcome Count: 6
harms Count: 1
qol Count: 0

outcomes

release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0000
source section id
NCT02395666-om-0000
source outcome index
0
endpoint
Number of Participants With Event Free Survival (EFS) During Study.
endpoint definition
To evaluate the preventative activity of DFMO as a single agent in patients that are in remission based on: Event free survival (EFS)
time origin
2 Years
analysis role
primary
reporting status
POSTED
data cutoff
Source null
population note
One subject removed from Stratum 1 due to not fitting study criteria upon review
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0001
source section id
NCT02395666-om-0001
source outcome index
1
endpoint
Percentage of Participants With Overall Survival (OS)
endpoint definition
To evaluate the preventative activity of DFMO as a single agent in patients with neuroblastoma who are in remission based on: Overall Survival (OS)
time origin
2 Years
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0002
source section id
NCT02395666-om-0002
source outcome index
2
endpoint
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
endpoint definition
To continue to determine the safety and tolerability of DFMO as a single agent and in pediatric and young adult patients with high risk neuroblastoma that is in remission.
time origin
2 years
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Stratum 1 and 2 were analyzed together as one safety group as per statistical analysis plan.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0003
source section id
NCT02395666-om-0003
source outcome index
3
endpoint
Test the Association of Survival With ODC1 Genotype
endpoint definition
Tests (p-value) of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype. Blood: microRNA analysis as predictor of DFMO effect, ornithine decarboxylase (ODC) single nucleotide polymorphism (SNP) analysis in DNA isolated from nucleated cells
time origin
2 years
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0004
source section id
NCT02395666-om-0004
source outcome index
4
endpoint
Peak Plasma Concentration (Cmax)
endpoint definition
Pharmacokinetic assay Cmax/D Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days.
time origin
Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
12 subjects from both Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0005
source section id
NCT02395666-om-0005
source outcome index
5
endpoint
Area Under the Plasma Concentration Versus Time Curve (AUC)
endpoint definition
Pharmacokinetic assay AUC(0-6 hr)/D Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days
time origin
0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose on two different days
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
12 subjects from both Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

arms

release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0000
arm key
OG000
arm label
Stratum 1
regimen
Subjects that are in remission at the end of upfront therapy defined as chemotherapy (5-7 cycles), surgery as indicated, consolidation therapy as indicated, radiation therapy as indicated, anti-GD2 antibody therapy with retinoic acid up to 6 cycles. Subjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.
analyzed count
100
analyzed unit
Participants
is comparator
Source null
source pointer
NCT02395666-om-0000#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0000
arm key
OG001
arm label
Stratum 2
regimen
Subjects that are in remission after any previous relapse or refractory therapy. Subjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.
analyzed count
39
analyzed unit
Participants
is comparator
Source null
source pointer
NCT02395666-om-0000#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0001
arm key
OG000
arm label
Stratum 1
regimen
Subjects that are in remission at the end of upfront therapy defined as chemotherapy (5-7 cycles), surgery as indicated, consolidation therapy as indicated, radiation therapy as indicated, anti-GD2 antibody therapy with retinoic acid up to 6 cycles. Subjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.
analyzed count
100
analyzed unit
Participants
is comparator
Source null
source pointer
NCT02395666-om-0001#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0001
arm key
OG001
arm label
Stratum 2
regimen
Subjects that are in remission after any previous relapse or refractory therapy. Subjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.
analyzed count
39
analyzed unit
Participants
is comparator
Source null
source pointer
NCT02395666-om-0001#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0002
arm key
OG000
arm label
DFMO Twice Daily
regimen
Subjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle. DFMO: Subjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.
analyzed count
140
analyzed unit
Participants
is comparator
Source null
source pointer
NCT02395666-om-0002#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0003
arm key
OG000
arm label
GG, GT, TT
regimen
Tests of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype.
analyzed count
140
analyzed unit
Participants
is comparator
Source null
source pointer
NCT02395666-om-0003#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0003
arm key
OG001
arm label
GG or GT, TT
regimen
Tests of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype.
analyzed count
140
analyzed unit
Participants
is comparator
Source null
source pointer
NCT02395666-om-0003#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0003
arm key
OG002
arm label
GG, GT or TT
regimen
Tests of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype.
analyzed count
140
analyzed unit
Participants
is comparator
Source null
source pointer
NCT02395666-om-0003#groups/OG002
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0004
arm key
OG000
arm label
Study Subjects Consented to PK Collection
regimen
Includes study subjects from either stratum that signed an optional consent to have PK's collected
analyzed count
12
analyzed unit
Participants
is comparator
Source null
source pointer
NCT02395666-om-0004#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
outcome key
om-0005
arm key
OG000
arm label
Study Subjects Consented to PK Collection
regimen
Includes study subjects from either stratum that signed an optional consent to have PK's collected
analyzed count
12
analyzed unit
Participants
is comparator
Source null
source pointer
NCT02395666-om-0005#groups/OG000

harms

release id
clinical-indications-draft-1
disease state id
ds-neuroblastoma
nct id
NCT02395666
harm key
EG000
source section id
NCT02395666-ae
event group key
EG000
event group label
All Study Subjects
serious affected
23
serious at risk
140
other affected
41
other at risk
140
frequency threshold
4
collection period
Through study completion plus 30 days, an average of 2 years.
event definition
All subjects that received at least one dose of DFMO. Since both arms received the same exact treatment, they were combined into one reporting group.
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

quality of life

setting source links

Preserved source evidence · Not medical advice