NCT04696029 · Giselle Sholler
DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma
What this study is about
Difluoromethylornithine (DFMO) will be used in an open label, conducted at multiple hospitals, study as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma.
View original scientific description
Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma.
Interventions
DRUG
Difluoromethylornithine
DFMO (difluoromethylornithine is an inhibitor of ornithine decarboxylase (ODC) designated chemically as 2-(difluoromethyl)-DL-ornithine monohydrochloride monohydrate. The dosage form to be used in this study is provided as a convex tablet containing 192 mg eflornithine (equivalent to 250 mg of eflornithine HCl, monohydrate). The tablets are packaged and sealed in opaque white HDPE bottles, and each bottle contains 100 tablets. The DMFO tablets are supplied by USWorldMeds (USWM). The tablets are to be stored at room temperature (20-250C).
Primary outcome measures
Number of participants with event free survival (EFS) during study
Time frame: 2 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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 pr
Where
- Little Rock, Arkansas
- Oakland, California
- San Diego, California
- Stanford, California
- Hartford, Connecticut
- Miami, Florida
- Orlando, Florida
- St. Petersburg, Florida
- Tampa, Florida
- Honolulu, Hawaii
- Chicago, Illinois
- Lexington, Kentucky
And 11 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 28, 2026 · Source of record for eligibility and locations