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Using DFMO to prevent recurrence in neuroblastoma patients in remission

NMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)

Phase 2 Interventional Milton S. Hershey Medical Center · NCT02679144

This study is testing if a medication called DFMO can help prevent neuroblastoma from coming back in patients who are in remission after their initial treatment.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment441 (estimated)
Ages1 Year to 30 Years
SexAll
SponsorMilton S. Hershey Medical Center Academic / other
Drugs / interventionsdinutuximab, chemotherapy, immunotherapy
Locations49 sites (Birmingham, Alabama and 48 other locations)
Trial IDNCT02679144 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the use of difluoromethylornithine (DFMO) as a maintenance therapy for patients with high-risk neuroblastoma who are in complete remission. Participants will receive DFMO orally for 730 days at specified dosages based on their treatment stratum. The study aims to evaluate the effectiveness of DFMO in preventing the recurrence of neuroblastoma after standard upfront therapy. It is an open-label, multicenter trial designed to gather data on the safety and efficacy of this treatment approach.

Who should consider this trial

Good fit: Ideal candidates are patients under 30 years old with a pathologically confirmed diagnosis of high-risk neuroblastoma who are currently in complete remission.

Not a fit: Patients who are not in complete remission or who have not undergone the required standard upfront therapy will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the risk of neuroblastoma recurrence in high-risk patients.

How similar studies have performed: While the use of DFMO in this context is novel, previous studies have shown promise in using similar maintenance therapies for cancer prevention.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* 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):

  1. No evidence of residual disease on scan
  2. 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:

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).

Exclusion Criteria:

* 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.

Where this trial is running

Birmingham, Alabama and 48 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Neuroblastoma
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.