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NeuroblastomaPhase 2NMTTNCT02679144

Neuroblastoma Maintenance Therapy Trial

A Phase 2 study testing Difluoromethylornithine (DFMO) in people with Neuroblastoma, run by Giselle Sholler at 47 sites in 2 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter, study for patients with neuroblastoma in remission. In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B)… Read more about this study

Who is it for?
Ages 1 year to 30 years
Conditions the study is for
Neuroblastoma
What is being tested?
Difluoromethylornithine (DFMO)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
441 participants
Where
47 sitesin 2 countries
Study dates
Feb 2016to Feb 2033
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 30 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • All patients must have a pathologically confirmed diagnosis of neuroblastoma, < 30.99 years of age and classified as high risk at the time of diagnosis. Exception: patients who are initially diagnosed as non-high-risk neuroblastoma, but later converted (and/or relapsed) to high risk neuroblastoma are also eligible.
  • All patients must be in complete remission (CR):
  • No evidence of residual disease on scan
  • No evidence of disease metastatic to bone marrow.
  • Specific Criteria by Stratum:
  • Stratum 1/1B: All patients must have completed standard upfront therapy that replicates treatment which patients who were enrolled on ANBL0032 received, including:
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  • intensive induction chemotherapy and (if feasible) resection of primary tumor, followed by: consolidation with high-dose chemotherapy with stem cell transplant and radiotherapy, followed by: immunotherapy with Ch14.18/IL-2/GM-CSF (dinutuximab) and retinoic acid;.
  • All subjects on Stratum 1/B must have also met the following criteria:
  • A pre-transplant disease status evaluation that met International Neuroblastoma Response Criteria (INRC) for CR (complete response), VGPR (very good partial response), or PR (partial response) for primary site, soft tissue metastases and bone metastases. Patients who meet those criteria must also meet the protocol-specified criteria for bone marrow response prior to transplant as outlined below: No more than 10% tumor involvement (based on total nucleated cellular content) seen on any specimen from a bilateral bone marrow aspirate/biopsy.
  • Stratum 2: Neuroblastoma that is in first complete remission following standard upfront therapy different from that described for Stratum 1.
  • Stratum 3: Neuroblastoma that failed to have a response of at least PR following induction chemotherapy and surgical resection of the primary tumor, but that has achieved CR following additional therapy.
  • Stratum 4: Patients who have achieved a second or subsequent CR following relapse(s).
  • Pre-enrollment tumor survey: Prior to enrollment on this study, a determination of mandatory disease staging must be performed:
  • Tumor imaging studies including
  • Bilateral bone marrow aspirates and biopsy
  • This disease assessment is required for eligibility and preferably should be done within 2 weeks prior to enrollment, but must be done within a maximum of 4 weeks before enrollment.
  • Timing from prior therapy:
  • Stratum 1/1B: Enrollment no later than 60 days after completion of upfront therapy, (last dose of cis-retinoic acid) with a maximum of 6 cycles of cis-retinoic acid maintenance therapy.
  • Stratum 2, 3 and 4: Enrollment no later than 60 days from last dose of the most recent therapy.
  • Patients must have a Lansky or Karnofsky Performance Scale score of > 50% and patients must have a life expectancy of ≥ 2 months.
  • All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated below.
  • Patients must have adequate organ functions at the time of registration:
  • Hematological: Total absolute phagocyte count ≥1000/μL
  • Liver: Subjects must have adequate liver function
  • Renal: Adequate renal function
  • Females of childbearing potential must have a negative pregnancy test. Patients of childbearing potential must agree to use an effective birth control method. Female patients who are lactating must agree to stop breast-feeding.
  • Written informed consent in accordance with institutional and FDA (food and drug administration) guidelines must be obtained from all subjects (or patients' legal representative).

Who cannot join

  • BSA (Body Surface Area) of <0.25 m2.
  • 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 hematological and bone marrow suppression effects of prior chemotherapy.
  • 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.

Trial locations and contacts

47 sites in 2 countries.

47 sites in 2 countries

United States41 sites

  • Augusta University Health

    Augusta, Georgia, 30912

  • Dell Children's Blood and Cancer Center

    Austin, Texas, 78723

  • University of Alabama, Children's of Alabama

    Birmingham, Alabama, 35233

  • St. Lukes

    Boise, Idaho, 83712

  • Tufts Medical Center

    Boston, Massachusetts, 02111

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • Levine Children's Hospital

    Charlotte, North Carolina, 28204

  • Advocate Aurora Research Institute

    Chicago, Illinois, 60453

  • Cleveland Clinic Children's

    Cleveland, Ohio, 44106

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

  • Children's Medical Center

    Dallas, Texas, 75235

  • Rocky Mountain Pediatric Hematology

    Denver, Colorado, 80205

  • Duke University

    Durham, North Carolina, 27708

  • University of Florida

    Gainesville, Florida, 32611

  • Helen DeVos Children's Hospital

    Grand Rapids, Michigan, 49503

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Connecticut Children's Hospital

    Hartford, Connecticut, 06106

  • Penn State Milton S. Hershey Medical Center and Children's Hospital

    Hershey, Pennsylvania, 17033

  • Kapiolani Medical Center for Women and Children

    Honolulu, Hawaii, 96813

  • Texas Children's Cancer and Hematology Centers

    Houston, Texas, 77030

  • University of Iowa

    Iowa City, Iowa, 52242

  • Children's Mercy Hospitals and Clinics

    Kansas City, Missouri, 64108

  • Kentucky Children's Hospital

    Lexington, Kentucky, 40536

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202

  • Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine

    Louisville, Kentucky, 40202

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Children's Hospital and Clinics of Minnesota

    Minneapolis, Minnesota, 55404

  • Monroe Carrell Jr. Children's Hospital at Vanderbilt

    Nashville, Tennessee, 37232

  • NYU Langone Health Hassenfeld Children's Hospital

    New York, New York, 10016

  • Children's Hospital of The King's Daughters

    Norfolk, Virginia, 23507

  • UCSF Benioff Children's Hospital Oakland-

    Oakland, California, 94609

  • Arnold Palmer Hospital for Children

    Orlando, Florida, 32806

  • Randall Children's Hospital

    Portland, Oregon, 97227

  • Hasbro Children's Hospital

    Providence, Rhode Island, 02903

  • Virginia Commonwealth University

    Richmond, Virginia, 23284

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113

  • Rady Children's Hospital

    San Diego, California, 92123

  • Cardinal Glennon Children's Medical Center

    St Louis, Missouri, 63104

  • All Children's Hospital Johns Hopkins Medicine

    St. Petersburg, Florida, 33701

  • St. Joseph's Children's Hospital

    Tampa, Florida, 33614

  • The Children's Hospital at Montefiore

    The Bronx, New York, 10467

Other trials in United States

Canada6 sites

  • Alberta Children's Hospital

    Calgary, Alberta, AB T3B 6A8

  • UHC Sainte-Justine

    Montreal, Quebec, QC H3S 2G4

  • Montreal Children's Hospital

    Montreal, Quebec, QC H4A 3H9

  • CHUQ

    Québec, Quebec, QC G1V 4W6

  • Janesway Children's Health and Rehabilitation Centre

    St. John's, Newfoundland and Labrador, NL A1B 3V6

  • CancerCare Manitoba

    Winnipeg, Manitoba, MB R3E 0V9

Other trials in Canada

About this study

Official title: NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)

Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter, study for patients with neuroblastoma in remission. In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study. This study will focus on the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to prevent recurrence.

Conditions
Neuroblastoma
Being tested
Difluoromethylornithine (DFMO)
Medical subject headings
Neuroblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Difluoromethylornithine (DFMO)Drug

    Also called eflornithine

    Subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study.

Study groups in NCT02679144
Study groupRoleWhat they receive
Difluoromethylornithine (DFMO)ExperimentalSubjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study.
Receives: Drug: Difluoromethylornithine (DFMO)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of participants with event free survival (EFS) during study.

    Measured: 2 years

Secondary outcomes (6)

  1. Length of time that participants experience Overall Survival (OS)

    Measured: 7 years

  2. Number of Participants with Adverse Events as a Measure of Safety and Tolerability

    Measured: 2 years

  3. Peak Plasma Concentration (Cmax)

    Pharmacokinetic assay

    Measured: 1 year

  4. Area under the plasma concentration versus time curve (AUC)

    Pharmacokinetic assay

    Measured: 1 year

  5. Time to reach Peak Plasma Concentration (Tmax)

    Pharmacokinetic assay

    Measured: 1 year

  6. Number of participants with ODC (Ornithine decarboxylase) single nucleotide polymorphisms.

    Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Feb 2016 (estimated)
  2. Study startFeb 2016
  3. Primary completionFeb 2028 (estimated)
  4. Study completionFeb 2033 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
441 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02679144
Sponsor's protocol ID
NMTRC014
Acronym
NMTT
Status last verified
Sep 2026
First submitted
4 Feb 2016
Last updated
3 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Duke ES, Bradford D, Sinha AK, Mishra-Kalyani PS, Lerro CC, Rivera D, Wearne E, Miller CP, Leighton J, Sabit H, Zhao H, Lane A, Scepura B, Pazdur R, Singh H, Kluetz PG, Donoghue M, Drezner N. US Food and Drug Administration Approval Summary: Eflornithine for High-Risk Neuroblastoma After Prior Multiagent, Multimodality Therapy. J Clin Oncol. 2024 Sep 1;42(25):3047-3057. doi: 10.1200/JCO.24.00546. Epub 2024 Jun 25.

    PubMed 38917371

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Difluoromethylornithine (DFMO) in people with Neuroblastoma, run by Giselle Sholler at 47 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 27 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 47 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with event free survival (EFS) during study. (time frame: 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02679144, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2026.

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