Recruiting

DFMO Maintenance Therapy for High Risk and Relapsed Medulloblastoma

0 criteria met from your profileSee at a glance how your profile meets each eligibility criteria.
Study Aim

This study aims to evaluate the effectiveness of difluoromethylornithine (DFMO) as a single treatment in preventing relapse in patients with high risk and very high risk medulloblastoma, as well as those with relapsed/refractory medulloblastoma, by observing the number of participants with event free survival during the study.

What is being tested

Difluoromethylornithine

Drug
Who is being recruted

Glioma+7

+ Medulloblastoma

+ Neoplasms

Until 21 Years
+20 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 2
Interventional
Study Start: March 2021
See protocol details

Summary

Principal SponsorGiselle Sholler
Study ContactBCC Enroll
Last updated: February 4, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: March 29, 2021

Actual date on which the first participant was enrolled.

This study focuses on a specific type of brain tumor called medulloblastoma, which is considered high risk or very high risk based on its molecular features, or has returned after treatment (relapsed) or not responded to treatment (refractory). The study is testing a drug called difluoromethylornithine (DFMO) as a maintenance therapy to prevent the tumor from growing or coming back. The goal is to improve treatment outcomes for this serious condition, potentially offering a new approach to manage high-risk medulloblastoma. Participants in this study will take DFMO orally, twice a day, for a total of 730 days. They will be divided into three groups based on the risk level or status of their medulloblastoma. The study aims to enroll 118 participants in total, ensuring that there will be 107 evaluable participants. The main outcome measured is the number of participants who remain event-free, meaning their tumor does not grow or return, during the study. This will help evaluate the effectiveness of DFMO in preventing relapse compared to historical controls.

Official TitlePhase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma
NCT04696029
Principal SponsorGiselle Sholler
Study ContactBCC Enroll
Last updated: February 4, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

118 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Until 21 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

GliomaMedulloblastomaNeoplasmsNeoplasms by Histologic TypeNeoplasms, Germ Cell and EmbryonalNeoplasms, Glandular and EpithelialNeoplasms, Nerve TissueNeuroectodermal TumorsNeuroectodermal Tumors, PrimitiveNeoplasms, Neuroepithelial

Criteria

16 inclusion criteria required to participate
MYC amplified Group 3
Non-WNT, non-SHH infant (< 3 yrs)
Age: 0-21 years of age at diagnosis
All patients must either have a pathologically confirmed diagnosis of medulloblastoma with molecular grouping identified by either Nanostring or methylation profiling.
Show More Criteria
4 exclusion criteria prevent from participating
BSA of <0.25 m2
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 are currently receiving another investigational drug are excluded from participation.
Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

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

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 23 locations

Recruiting

Arkansas Children's Hospital

Little Rock, United StatesOpen Arkansas Children's Hospital in Google Maps
Recruiting

UCSF Benioff Children's Hospital Oakland

Oakland, United States
Recruiting

Rady Children's Hospital

San Diego, United States
Recruiting

Stanford University

Stanford, United States
Recruiting
23 Study Centers