Trial · NCT02395666

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

NCT02395666, Phase 2, Completed, Giselle Sholler; results first posted 2020-04-03

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission. Trials, record NCT02395666. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02395666

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission
NCT number: NCT02395666
Status: Completed
Phases: Phase 2
Enrollment: 140 participants, actual
Lead sponsor: Giselle Sholler
Conditions: Neuroblastoma
Interventions: DFMO
First posted: 2015-03-23
Last updated: 2024-08-06
Results first posted: 2020-04-03

Source ClinicalTrials.gov

Registered outcomes7

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Event Free Survival (EFS) During Study.	2 Years	To evaluate the preventative activity of DFMO as a single agent in patients that are in remission based on: Event free survival (EFS)
Secondary	Percentage of Participants With Overall Survival (OS)	2 Years	To evaluate the preventative activity of DFMO as a single agent in patients with neuroblastoma who are in remission based on: Overall Survival (OS)
Secondary	Number of Participants With Adverse Events as a Measure of Safety and Tolerability	2 years	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.
Secondary	Test the Association of Survival With ODC1 Genotype	2 years	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
Secondary	Peak Plasma Concentration (Cmax)	Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days	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.
Secondary	Area Under the Plasma Concentration Versus Time Curve (AUC)	0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose on two different days	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
Secondary	Time to Reach Peak Plasma Concentration (Tmax)	0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose on two different days	Pharmacokinetic assay- tmax, hr Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days

Source ClinicalTrials.gov

Outcomes7

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Event Free Survival (EFS) During Study.	2 Years	One subject removed from Stratum 1 due to not fitting study criteria upon review	percentage of subjects without an event	MEAN
SECONDARY	Percentage of Participants With Overall Survival (OS)	2 Years		percentage of subjects without an event	MEAN
SECONDARY	Number of Participants With Adverse Events as a Measure of Safety and Tolerability	2 years	Stratum 1 and 2 were analyzed together as one safety group as per statistical analysis plan.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Test the Association of Survival With ODC1 Genotype	2 years	Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.	p-value	NUMBER
SECONDARY	Peak Plasma Concentration (Cmax)	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.	ng/mL	MEAN
SECONDARY	Area Under the Plasma Concentration Versus Time Curve (AUC)	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.	hr*ng/mL	MEAN
SECONDARY	Time to Reach Peak Plasma Concentration (Tmax)	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.	hours	MEAN

Source ClinicalTrials.gov

Results by group11

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Number of Participants With Event Free Survival (EFS) During Study.	Stratum 1	84	95% Confidence Interval 80 to 88	percentage of subjects without an event
Number of Participants With Event Free Survival (EFS) During Study.	Stratum 2	51	95% Confidence Interval 43 to 59	percentage of subjects without an event
Percentage of Participants With Overall Survival (OS)	Stratum 1	97	95% Confidence Interval 95 to 99	percentage of subjects without an event
Percentage of Participants With Overall Survival (OS)	Stratum 2	84	95% Confidence Interval 78 to 90	percentage of subjects without an event
Number of Participants With Adverse Events as a Measure of Safety and Tolerability	DFMO Twice Daily	57		Participants
Test the Association of Survival With ODC1 Genotype	GG, GT, TT	0.96		p-value
Test the Association of Survival With ODC1 Genotype	GG or GT, TT	0.58		p-value
Test the Association of Survival With ODC1 Genotype	GG, GT or TT	0.67		p-value
Peak Plasma Concentration (Cmax)	Study Subjects Consented to PK Collection	11958	90% Confidence Interval 5123 to 18793	ng/mL
Area Under the Plasma Concentration Versus Time Curve (AUC)	Study Subjects Consented to PK Collection	47024	90% Confidence Interval 23868 to 70180	hr*ng/mL

1 to 10 of 11Source ClinicalTrials.gov

See all 11 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Stratum 1	101
Overall Study	STARTED	Stratum 2	39
Overall Study	COMPLETED	Stratum 1	95
Overall Study	COMPLETED	Stratum 2	38
Overall Study	NOT COMPLETED	Stratum 1	6
Overall Study	NOT COMPLETED	Stratum 2	1

Source ClinicalTrials.gov

Adverse events25

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Neutrophil count decrease	Blood and lymphatic system disorders	All Study Subjects	11	140	11
other	Anemia	Blood and lymphatic system disorders	All Study Subjects	6	140	6
other	ALT elevation	Hepatobiliary disorders	All Study Subjects	14	140	14
other	AST elevation	Hepatobiliary disorders	All Study Subjects	10	140	10
other	Diarrhea	Gastrointestinal disorders	All Study Subjects	9	140	9
other	Hearing Loss	Ear and labyrinth disorders	All Study Subjects	9	140	9
other	Otitis Media	Infections and infestations	All Study Subjects	6	140	6
serious	Infection	Infections and infestations	All Study Subjects	8	140	8
serious	Vomiting	Gastrointestinal disorders	All Study Subjects	3	140	3
serious	Pain	General disorders	All Study Subjects	1	140	1

1 to 10 of 25Source ClinicalTrials.gov

See all 25 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	All Study Subjects	0	140
Total, other adverse events	All Study Subjects	41	140
Total, serious adverse events	All Study Subjects	23	140

Source ClinicalTrials.gov

Papers5

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
32391579	DERIVED	PubMed
32506485	DERIVED	PubMed
34522028	DERIVED	PubMed
37883734	DERIVED	PubMed
38917371	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT02395666

GET https://conductscience.org/api/trials/v1/NCT02395666/events?page=2

API reference For AI assistants