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New hope for kids with aggressive brain cancer: drug aims to stop relapse

NCT ID NCT04696029

Disease control Sponsor: Giselle Sholler Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 13, 2026 · Updated 2 times

Summary

This study tests a drug called DFMO (eflornithine) as a maintenance therapy to prevent medulloblastoma, a type of brain cancer, from returning in children. It includes 118 participants aged 0-21 with high-risk or relapsed forms of the disease. The goal is to see if DFMO can extend the time without cancer progression compared to past outcomes.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 118 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2021

Expected to finish

Mar 2029

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

medulloblastoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Medulloblastoma

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    26 sites in 2 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Advocate Aurora Research Institute

    RECRUITING

    Chicago, Illinois, 60453, United States

  • All Children's Hospital Johns Hopkins Medicine

    RECRUITING

    St. Petersburg, Florida, 33701, United States

  • Arkansas Children's Hospital

    RECRUITING

    Little Rock, Arkansas, 72202, United States

  • Arnold Palmer Hospital for Children

    RECRUITING

    Orlando, Florida, 32806, United States

  • CHUQ

    RECRUITING

    Québec, Quebec, QC G1V 4W6, Canada

  • Cardinal Glennon Children's Medical Center

    RECRUITING

    St Louis, Missouri, 63104, United States

  • Children's Mercy Hospitals and Clinics

    RECRUITING

    Kansas City, Missouri, 64108, United States

  • Connecticut Children's Hospital

    RECRUITING

    Hartford, Connecticut, 06106, United States

  • Dell Children's Blood and Cancer Center

    RECRUITING

    Austin, Texas, 78723, United States

  • Duke University

    RECRUITING

    Durham, North Carolina, 27708, United States

  • Hackensack University Medical Center

    RECRUITING

    Hackensack, New Jersey, 07601, United States

  • Hasbro Children's Hospital

    RECRUITING

    Providence, Rhode Island, 02903, United States

  • Kapiolani Medical Center for Women and Children

    RECRUITING

    Honolulu, Hawaii, 96813, United States

  • Kentucky Children's Hospital

    RECRUITING

    Lexington, Kentucky, 40502, United States

  • Levine Children's Hospital

    RECRUITING

    Charlotte, North Carolina, 28204, United States

  • Medical University of South Carolina

    RECRUITING

    Charleston, South Carolina, 29425, United States

  • Nicklaus Children's Hospital

    RECRUITING

    Miami, Florida, 33155, United States

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

    RECRUITING

    Louisville, Kentucky, 40202, United States

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

    RECRUITING

    Hershey, Pennsylvania, 17033, United States

  • Rady Children's Hospital

    RECRUITING

    San Diego, California, 92123, United States

  • Randall Children's Hospital

    RECRUITING

    Portland, Oregon, 97227, United States

  • St. Joseph's Children's Hospital

    RECRUITING

    Tampa, Florida, 33607, United States

  • Stanford University

    RECRUITING

    Stanford, California, 94305, United States

  • UC Davis Comprehensive Cancer Center

    RECRUITING

    Sacramento, California, 95817, United States

  • UCSF Benioff Children's Hospital Oakland

    RECRUITING

    Oakland, California, 94609, United States

  • University of Oklahoma

    RECRUITING

    Oklahoma City, Oklahoma, 73104, United States

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