---
title: Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission
nct_id: NCT02395666
overall_status: COMPLETED
phase: PHASE2
sponsor: Giselle Sholler
study_type: INTERVENTIONAL
primary_condition: Neuroblastoma
countries: United States
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT02395666.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT02395666"
ct_last_update_post_date: 2024-08-06
last_seen_at: "2026-05-12T06:22:22.085Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission

**Official Title:** A Phase II Preventative Trial of DFMO (Eflornithine HCl) as a Single Agent in Patients With High Risk Neuroblastoma in Remission

**NCT ID:** [NCT02395666](https://clinicaltrials.gov/study/NCT02395666)

## Key Facts

- **Status:** COMPLETED
- **Phase:** PHASE2
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 140
- **Lead Sponsor:** Giselle Sholler
- **Collaborators:** Beat NB Cancer Foundation, Because of Ezra, K C Pharmaceuticals Inc.
- **Conditions:** Neuroblastoma
- **Start Date:** 2015-03-05
- **Completion Date:** 2023-08-24
- **CT.gov Last Update:** 2024-08-06

## Brief Summary

The purpose of this research study is to evaluate a new investigational drug to prevent reoccurrence of neuroblastoma that is in remission. This study drug is called DFMO. The objectives of this study will be to monitor for safety and look at efficacy of DFMO.

The safety of the proposed dosing regimen in this trial will be tested by an on-going risk/benefit assessment during the study. A patient benefiting from treatment, not progressing on therapy, and in the absence of any safety issues associated with DFMO may continue on treatment up to 27 cycles with the expectation that there will be an overall clinical benefit.

The procedures involved in this study include Medical history, Physical exam, Vital signs (blood pressure, pulse, temperature), Blood tests, Urine tests, MRI or CT scan of the tumor(s), meta-iodobenzylguanidine (MIBG) scans, and Bone marrow aspirations. All of these tests and procedures are considered standard of care for this population. Drug administration is also part of this protocol, including an investigational new drug called DFMO.

The proposed dosing regimen is an oral dose of DFMO tablets two times a day for each day while on study. There will be 27 cycles. Each cycle will be 28 days in length.

## Eligibility

- **Maximum age:** 21 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
Inclusion Criteria:

* Age: 0-21 years at the time of diagnosis.
* Diagnosis: histologic verification at either the time of original diagnosis or a previous relapse of high risk neuroblastoma.
* Disease Status: Neuroblastoma that is in remission
* First dose of study medication must be greater than 30 days from completion of cytotoxic and antibody therapy and less than 120 days from previous therapy
* A negative serum or urine pregnancy test is required for female subjects of child bearing potential (onset of menses or ≥13 years of age).
* Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrel implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended.
* Absolute Neutrophil Count (ANC) \> 500/μl and platelet count \>50,000/μl
* Organ Function Requirements: Subjects must have adequate liver function as defined by:

  * Aspartate Aminotransferase (AST) and Alanine transaminase (ALT) \<10x upper limit of normal
  * Serum bilirubin must be ≤ 2.0 mg/dl
  * Serum creatinine based on age/gender
* Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines

Exclusion Criteria:

* Lansky score \< 60%
* Body Surface Area (BSA) (m2) of \<0.25
* Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
* Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the effects of prior chemotherapy (hematological and bone marrow suppression effects).
* Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
* Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.
```

## Arms

- **DFMO twice daily** (EXPERIMENTAL) — 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.

## Interventions

- **DFMO** (DRUG) — 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.

## Primary Outcomes

- **Number of Participants With Event Free Survival (EFS) During Study.** _(time frame: 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 Outcomes

- **Percentage of Participants With Overall Survival (OS)** _(time frame: 2 Years)_
- **Number of Participants With Adverse Events as a Measure of Safety and Tolerability** _(time frame: 2 years)_
- **Test the Association of Survival With ODC1 Genotype** _(time frame: 2 years)_
- **Peak Plasma Concentration (Cmax)** _(time frame: Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days)_
- **Area Under the Plasma Concentration Versus Time Curve (AUC)** _(time frame: 0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose on two different days)_
- **Time to Reach Peak Plasma Concentration (Tmax)** _(time frame: 0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose on two different days)_

## Locations (22)

- Phoenix Children's Hospital, Phoenix, Arizona, United States
- Arkansas Children's Hospital, Little Rock, Arkansas, United States
- Rady Children's Hospital, San Diego, California, United States
- Connecticut Children's Hospital, Hartford, Connecticut, United States
- Arnold Palmer Hospital for Children, Orlando, Florida, United States
- All Children's Hospital Johns Hopkins Medicine, St. Petersburg, Florida, United States
- Kapiolani Medical Center for Women and Children, Honolulu, Hawaii, United States
- Tufts Medical Center, Boston, Massachusetts, United States
- Helen DeVos Children's Hospital, Grand Rapids, Michigan, United States
- Children's Hospital and Clinics on Minnesota, Minneapolis, Minnesota, United States
- Children's Mercy Hospitals and Clinics, Kansas City, Missouri, United States
- Cardinal Glennon Children's Medical Center, St Louis, Missouri, United States
- The Children's Hospital at Montefiore, The Bronx, New York, United States
- Levine Children's Hospital, Charlotte, North Carolina, United States
- Nationwide Children's Hospital, Columbus, Ohio, United States
- Penn State Milton S. Hershey Medical Center and Children's Hospital, Hershey, Pennsylvania, United States
- Medical University of South Carolina, Charleston, South Carolina, United States
- Monroe Carrell Jr. Children's Hospital at Vanderbilt, Nashville, Tennessee, United States
- Dell Children's Blood and Cancer Center, Austin, Texas, United States
- Children's Medical Center, Dallas, Texas, United States
- Texas Children's Cancer and Hematology Centers, Houston, Texas, United States
- Primary Children's Hospital, Salt Lake City, Utah, United States

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `sponsor.collaborators` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.phoenix children's hospital|phoenix|arizona|united states` — added _(2026-05-12)_
- `locations.arkansas children's hospital|little rock|arkansas|united states` — added _(2026-05-12)_
- `locations.rady children's hospital|san diego|california|united states` — added _(2026-05-12)_
- `locations.connecticut children's hospital|hartford|connecticut|united states` — added _(2026-05-12)_
- `locations.arnold palmer hospital for children|orlando|florida|united states` — added _(2026-05-12)_
- `locations.all children's hospital johns hopkins medicine|st. petersburg|florida|united states` — added _(2026-05-12)_
- `locations.kapiolani medical center for women and children|honolulu|hawaii|united states` — added _(2026-05-12)_
- `locations.tufts medical center|boston|massachusetts|united states` — added _(2026-05-12)_
- `locations.helen devos children's hospital|grand rapids|michigan|united states` — added _(2026-05-12)_
- `locations.children's hospital and clinics on minnesota|minneapolis|minnesota|united states` — added _(2026-05-12)_
- `locations.children's mercy hospitals and clinics|kansas city|missouri|united states` — added _(2026-05-12)_
- `locations.cardinal glennon children's medical center|st louis|missouri|united states` — added _(2026-05-12)_
- `locations.the children's hospital at montefiore|the bronx|new york|united states` — added _(2026-05-12)_
- `locations.levine children's hospital|charlotte|north carolina|united states` — added _(2026-05-12)_
- `locations.nationwide children's hospital|columbus|ohio|united states` — added _(2026-05-12)_
- `locations.penn state milton s. hershey medical center and children's hospital|hershey|pennsylvania|united states` — added _(2026-05-12)_
- `locations.medical university of south carolina|charleston|south carolina|united states` — added _(2026-05-12)_
- `locations.monroe carrell jr. children's hospital at vanderbilt|nashville|tennessee|united states` — added _(2026-05-12)_
- `locations.dell children's blood and cancer center|austin|texas|united states` — added _(2026-05-12)_
- `locations.children's medical center|dallas|texas|united states` — added _(2026-05-12)_
- `locations.texas children's cancer and hematology centers|houston|texas|united states` — added _(2026-05-12)_
- `locations.primary children's hospital|salt lake city|utah|united states` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT02395666.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT02395666*  
*This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*