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Using DFMO for Maintenance Therapy in High Risk Medulloblastoma

Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma

Phase 2 Interventional Milton S. Hershey Medical Center · NCT04696029

This study is testing if a new oral medication called DFMO can help children and young adults with high-risk medulloblastoma stay cancer-free after their initial treatment.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment118 (estimated)
AgesN/A to 21 Years
SexAll
SponsorMilton S. Hershey Medical Center Academic / other
Drugs / interventionschemotherapy, radiation
Locations24 sites (Little Rock, Arkansas and 23 other locations)
Trial IDNCT04696029 on ClinicalTrials.gov

What this trial studies

This study evaluates the use of difluoromethylornithine (DFMO) as a maintenance therapy for children and young adults with molecular high risk, very high risk, and relapsed medulloblastoma. Participants will receive DFMO orally for 730 days at a dose of 2500 mg/m2 twice daily. The study will enroll a total of 118 subjects across three cohorts, each defined by specific molecular risk factors associated with their medulloblastoma diagnosis. The goal is to assess the efficacy and safety of DFMO in these high-risk populations.

Who should consider this trial

Good fit: Ideal candidates are children and young adults aged 0-21 with a confirmed diagnosis of medulloblastoma and specific molecular risk factors.

Not a fit: Patients with low-risk medulloblastoma or those who do not meet the molecular criteria for the cohorts may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve survival rates and outcomes for patients with high-risk medulloblastoma.

How similar studies have performed: While the use of DFMO in this context is novel, previous studies have explored its potential in other cancers, indicating a promising avenue for further investigation.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Age: 0-21 years of age at diagnosis
2. Pathology All patients must either have a pathologically confirmed diagnosis of medulloblastoma with molecular grouping identified by either Nanostring or methylation profiling.

   Cohort 1- Molecular High Risk:
   * Metastatic non-MYC amplified Group 3
   * Metastatic Group 4
   * Metastatic non-WNT/non-SHH (Must be non-MYC amplified)

   Cohort 2- Molecular Very High Risk
   * Metastatic OR MYCN amplified OR TP53 mutant non-infant (\>3 yrs) SHH
   * MYC amplified Group 3
   * Non-WNT, non-SHH infant (\< 3 yrs)

   Cohort 3: Relapsed/Refractory Medulloblastoma
3. Pre-enrollment tumor survey:

   Prior to enrollment on this study, a determination of mandatory disease staging must be performed:
   * Tumor imaging studies including: Brain and spine MRI
   * Lumbar Puncture only if previously positive
   * Bone Marrow aspiration/biopsy only if previously positive
   * This disease assessment is required for eligibility and preferably should be done within 2 weeks prior to first dose of study drug, but must be done within a maximum of 4 weeks before first dose of study drug.
4. Disease Status: Subjects must have no evidence of disease, or stable\* residual nonbulky\*\* disease.

   \*Stable residual disease defined as non-progression over 2 separate imaging studies at least 6 weeks apart

   \*\*Non-bulky disease defined as maximal cross-sectional area \< 3cm\^2 at enrollment. Patients with leptomeningeal disease are allowed to participate on study.
5. Timing from prior therapy:

   Enrollment (first dose of DFMO) no later than 60 days after last dose of conventional chemotherapy. Patients who have undergone high dose chemotherapy (HDCT) with autologous stem cell transplantation (SCT) are eligible if more than 45 days have elapsed since date of last SCT.
6. Patients must have a Lansky or Karnofsky Performance Scale score of ≥ 50% (see Appendix II) and patients must have a life expectancy of ≥ 2 months.
7. All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to first dose of study drug unless otherwise indicated below.
8. Patients must have adequate organ functions at the time of registration:

   * Hematological: Hematological recovery as defined by ANC ≥750/μL, platelets ≥30 (non-transfused x 7 days)
   * Liver: Adequate liver function as defined by AST and ALT \<10x upper limit of normal
   * Renal: Adequate renal function defined as (perform one of the following): Creatinine clearance or radioisotope GFR ≥ 70 mL/min/1.73 m2 or a serum creatinine based on age/gender
9. 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.
10. Written informed consent in accordance with institutional and FDA guidelines must be obtained from all subjects (or patients' legal representative).

Exclusion Criteria:

1. BSA of \<0.25 m2
2. Metastatic disease outside of CNS
3. Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment
4. Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
5. Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.
6. 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.
7. 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

Little Rock, Arkansas and 23 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 Medulloblastoma
Last reviewed 2026-06-09 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.