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DFMO for Medulloblastoma

This study is testing a drug called difluoromethylornithine (DFMO) as a maintenance therapy for medulloblastoma, a type of brain cancer. DFMO is given as a tablet. The study is for children and young adults up to 21 years old who have medulloblastoma with specific molecular features (like high/very high risk or if the cancer has returned). Researchers want to see how many participants are free from events (like the cancer coming back) over 2 years, with a 5-year follow-up. The study aims to enroll 118 participants.

Study design
This is an open-label study, meaning both you and your doctors will know you are receiving DFMO. It plans to enroll 118 participants across three groups based on their medulloblastoma type.
What's involved
You would take oral DFMO twice a day for 730 days (about two years).
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 5 years after the 2-year study period to track their event-free survival.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04696029

DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma

Recruiting
PHASE2Up to 21InterventionalTreatment
Giselle Sholler
~118 participants
Updated 2026-08-12 on ClinicalTrials.gov
What's tested:Difluoromethylornithine

At a glance

Recruiting sites
26 of 26 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of participants with event free survival (EFS) during study
Measured over 2 years plus 5 years follow up

Conditions

Medulloblastoma

Explore related trials

Medulloblastoma

NCT04696029

Where you'd take part

This study runs at 26 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Advocate Aurora Research Institute

    Chicago, Illinoisstudy coordinator listed

    Recruiting

  • All Children's Hospital Johns Hopkins Medicine

    St. Petersburg, Floridastudy coordinator listed

    Recruiting

  • Arkansas Children's Hospital

    Little Rock, Arkansasstudy coordinator listed

    Recruiting

  • Arnold Palmer Hospital for Children

    Orlando, Floridastudy coordinator listed

    Recruiting

  • Cardinal Glennon Children's Medical Center

    St Louis, Missouristudy coordinator listed

    Recruiting

  • Children's Mercy Hospitals and Clinics

    Kansas City, Missouristudy coordinator listed

    Recruiting

  • CHUQ

    Québec, Quebec, Canadastudy coordinator listed

    Recruiting

  • Connecticut Children's Hospital

    Hartford, Connecticutstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Michael A Huang, MD · STUDY_CHAIR · Beat Childhood Cancer

Who to contact

BCC Enroll
[email protected]
7175310003
Email the study team

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Eligibility criteria

Inclusion

Metastatic non-MYC amplified Group 3
Metastatic Group 4
Metastatic non-WNT/non-SHH (Must be non-MYC amplified)
Metastatic OR MYCN amplified OR TP53 mutant non-infant (\>3 yrs) SHH
MYC amplified Group 3
Non-WNT, non-SHH infant (\< 3 yrs)
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.
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).

What this trial measures

  • Number of participants with event free survival (EFS) during study2 years plus 5 years follow up

    o To evaluate the efficacy of difluoromethylornithine (DFMO) as a single agent in preventing relapse in patients with molecular high risk and very high risk medulloblastoma, and relapsed/refractory medulloblastoma based upon the 2-year progression-free survival rate (PFS) compared to relevant historical controls.