Taking people in CanadaPhase 2

DFMO as ongoing treatment for medulloblastoma that is high risk or has returned

Tests DFMO as maintenance treatment in young people with high-risk or relapsed medulloblastoma.

Plain-language title generated from the registry record
Registry title: DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma

Key facts

Where: Québec, CanadaSee the site
Phase: Phase 2

AI summary: who the study is looking for

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for children and young people with medulloblastoma that is high risk, very high risk, or has come back or not responded. They take DFMO as maintenance treatment.

Age: 0 to 21 years old at diagnosis.
Sex: All sexes can join.
Disease stage: Medulloblastoma confirmed by pathology with a molecular group. High risk or very high risk groups, or relapsed/refractory disease. No evidence of disease, or stable residual disease that is not bulky (under 3cm (1.2 in)^2).
Earlier treatment: First dose of DFMO no later than 60 days after the last conventional chemotherapy. After high dose chemotherapy with stem cell transplant, more than 45 days must have passed since the transplant.
Biomarker or test result: Molecular grouping must be identified by Nanostring or methylation profiling.

Main reasons for not taking part

Body surface area under 0.25 m2
Metastatic disease outside the central nervous system
Relapsed/refractory patients aged 5 years or older who have never had radiation
Currently receiving another investigational drug or other anticancer agents
Uncontrolled infection

Read by machine on 12 November 2026.

Where in Canada

CHUQQuébec, CanadaContact: Valérie-Ève JulienTaking people

Questions to ask the study team

How often do I need to come in, and for how long each time?
Are travel costs paid?
Can I keep my own doctor?
What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Trial details

Who runs it: Giselle Sholler
Trial code: NCT04696029
Record updated: 12 August 2026
Original 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.Read full original descriptionShow less
Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma
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.
Inclusion: Age: 0-21 years of age at diagnosis; 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; 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; 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; 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; 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; 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; 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; 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 guidelines must be obtained from all subjects (or patients' legal representative).
Exclusion: 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 the hematological and bone marrow suppression effects of prior chemotherapy; Metastatic disease outside of CNS; 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; Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment; BSA of <0.25 m2; 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.
ClinicalTrials.gov NCT04696029