AzTrials
AzTrials

MedulloblastomaPhase 2NCT04696029

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

A Phase 2 study testing Difluoromethylornithine in people with Medulloblastoma, run by Giselle Sholler at 26 sites in 2 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

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 more about this study

Who is it for?
Ages Up to 21 years
Conditions the study is for
Medulloblastoma
What is being tested?
Difluoromethylornithine
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
118 participants
Where
26 sitesin 2 countries
Study dates
29 Mar 2021to Mar 2029
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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)
Show all 30Show fewer
  • 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).

Who cannot join

  • BSA of <0.25 m2
  • Metastatic disease outside of CNS
  • Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment
  • 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.
  • 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.

Trial locations and contacts

26 sites in 2 countries. Contact the study team or a site near you to ask about taking part.

BCC Enroll contact

7175310003

BCCEnroll@pennstatehealth.psu.edu

26 sites in 2 countries

United States25 sites, 25 recruiting

  • Dell Children's Blood and Cancer Center

    Austin, Texas, 78723

    Rhea Robinson, RN, rmrobinson@ascension.org

    Virginia Harrod

    Recruiting

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

    Shanta Salzar, MD, salzers@musc.edu

    Jaqueline Kraveka

    Recruiting

  • Levine Children's Hospital

    Charlotte, North Carolina, 28204

    Jontyce Green, Jontyce.Green@advocatehealth.org

    Thomas Russell

    Recruiting

  • Advocate Aurora Research Institute

    Chicago, Illinois, 60453

    Jennifer Ward, jennifer.ward@aah.org

    Rebecca McFall

    Recruiting

  • Duke University

    Durham, North Carolina, 27708

    Morgan Low, morgan.low@duke.edu

    Jessica Sun

    Recruiting

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

    Kellie Daniel, kellie.danielle@hmhn.org

    Derek Hanson

    Recruiting

  • Connecticut Children's Hospital

    Hartford, Connecticut, 06106

    Adam Barselau, Abarselau@connecticutchildrens.org

    Michael Isakoff

    Recruiting

  • Penn State Milton S. Hershey Medical Center and Children's Hospital

    Hershey, Pennsylvania, 17033

    Penn State Clinical Trials, ExtractClinicalTrials@pennstatehealth.psu.edu

    Valerie Brown

    Recruiting

  • Kapiolani Medical Center for Women and Children

    Honolulu, Hawaii, 96813

    Andrea Siu, andrea.siu@kapiolani.org

    Kelley Hutchins

    Recruiting

  • Children's Mercy Hospitals and Clinics

    Kansas City, Missouri, 64108

    Nicole Harvey, ndharvey@cmh.edu

    Kevin Ginn

    Recruiting

  • Kentucky Children's Hospital

    Lexington, Kentucky, 40502

    Brittany Fuller, blfull2@email.uky.edu

    Tom Badgett

    Recruiting

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202

    Susan Hall, HallSF@archildrens.org

    Kevin Bielamowicz

    Recruiting

  • Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine

    Louisville, Kentucky, 40202

    Jennifer Miller

    Mustafa Barbour

    Recruiting

  • Nicklaus Children's Hospital

    Miami, Florida, 33155

    Aixa Guadarrama, Aixa.Guadarrama@Nicklaushealth.org

    Ossama Maher

    Recruiting

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609

    Group Contact, PedOncRschOAK@ucsf.edu

    Caroline Hastings

    Recruiting

  • University of Oklahoma

    Oklahoma City, Oklahoma, 73104

    Christina Gonzalez, Christina-gonzalez@ou.edu

    Rene McNall-Knapp

    Recruiting

  • Arnold Palmer Hospital for Children

    Orlando, Florida, 32806

    Marie Frankos, marie.frankos@orlandohealth.com

    Jamie Libes-Bander

    Recruiting

  • Randall Children's Hospital

    Portland, Oregon, 97227

    Aaron White, AJWHITE@lhs.org

    Jason Glover

    Recruiting

  • Hasbro Children's Hospital

    Providence, Rhode Island, 02903

    Christopher Bouressa, cbouressa@lifespan.org

    Bradley DeNardo

    Recruiting

  • UC Davis Comprehensive Cancer Center

    Sacramento, California, 95817

    Mary Gay Lapasaran, mlapasaran@health.ucdavis.edu

    Marcio Malogolowkin

    Recruiting

  • Rady Children's Hospital

    San Diego, California, 92123

    Sarah Hollander, shollander@rchsd.org

    William Roberts

    Recruiting

  • Cardinal Glennon Children's Medical Center

    St Louis, Missouri, 63104

    Gina Martin, gina.martin@health.slu.edu

    William Ferguson

    Recruiting

  • All Children's Hospital Johns Hopkins Medicine

    St. Petersburg, Florida, 33701

    Kevin Samnarine, ksamnar1@jh.edu

    Jennifer Dean

    Recruiting

  • Stanford University

    Stanford, California, 94305

    Stefania U Chirita, schirita@stanford.edu

    Raya Saab

    Recruiting

  • St. Joseph's Children's Hospital

    Tampa, Florida, 33607

    Jennifer Manns, Jennifer.Manns@baycare.org

    Don Eslin

    Recruiting

Other trials in United States

Canada1 site, 1 recruiting

  • CHUQ

    Québec, Quebec, QC G1V 4W6

    Valérie-Ève Julien, Valerie-Eve.Julien@crchudequebec.ulaval.ca

    Bruno Michon

    Recruiting

Other trials in Canada

About this study

Official title: 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.

Read the detailed description

In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 2500 mg/m2 BID on each day of study.

Subjects will be evaluated in 3 Cohorts:

Cohort 1: Molecular High Risk Medulloblastoma Cohort 2: Molecular Very High Risk Medulloblastoma Cohort 3: Relapsed/Refractory Medulloblastoma

A total of 118 subjects across all cohorts will be enrolled to ensure that there will be 107 evaluable subjects (32-39 per cohort)

Conditions
Medulloblastoma
Being tested
Difluoromethylornithine
Medical subject headings
Medulloblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DifluoromethylornithineDrug

    Also called Eflornithine, DFMO

    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).

Study groups in NCT04696029
Study groupRoleWhat they receive
Difluoromethylornithine (DFMO)Experimentalstudy subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 2500 mg/m2 BID on each day of study.
Receives: Drug: Difluoromethylornithine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of participants with event free survival (EFS) during study

    • 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.

    Measured: 2 years plus 5 years follow up

Secondary outcomes (4)

  1. Length of time that participants experience Overall Survival (OS)

    • To evaluate the efficacy of difluoromethylornithine (DFMO) as a single agent in patients with molecular high risk and very high risk medulloblastoma, and relapsed/refractory medulloblastoma based upon overall survival

    Measured: 7 years

  2. Determine the Overall Response Rate (ORR) of Participants using Modified RANO Criteria

    To evaluate the efficacy of difluoromethylornithine (DFMO) as a single agent in patients with molecular high risk and very high risk medulloblastoma, and relapsed/refractory medulloblastoma based upon Response Rate for patients with non-bulky residual disease present.

    Measured: 2 years

  3. Number of Participants with Adverse Events as a Measure of Safety and Tolerability

    To develop a complete safety and tolerability profile of difluoromethylornithine (DFMO) in pediatric and young adult subjects with medulloblastoma.

    Measured: 2 years plus 30 days

  4. Determine amount of DFMO in the CSF at 3 hours post dose

    • To measure CSF penetration after DFMO administration in pediatric subjects with medulloblastoma

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Jan 2021
  2. Study start29 Mar 2021
  3. Primary completionMar 2028 (estimated)
  4. Study completionMar 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
118 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04696029
Sponsor's protocol ID
BCC016
Status last verified
Aug 2026
First submitted
4 Jan 2021
Last updated
12 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Difluoromethylornithine in people with Medulloblastoma, run by Giselle Sholler at 26 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 30 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 12 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 26 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact BCC Enroll (7175310003, BCCEnroll@pennstatehealth.psu.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with event free survival (EFS) during study (time frame: 2 years plus 5 years follow up). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04696029.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in