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DFMO for Medulloblastoma

Phase 2
Recruiting
Research Sponsored by Giselle Sholler
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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.
Cohort 1- Molecular High Risk: Metastatic non-MYC amplified Group 3, Metastatic Group 4, Metastatic non-WNT/non-SHH (Must be non-MYC amplified)
Must not have
Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment
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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years plus 5 years follow up
Awards & highlights
No Placebo-Only Group

Summary

This trial will test the use of DFMO as a maintenance therapy for patients with high risk or very high risk medulloblastoma, as well as those who have relapsed or are refractory to other treatments.

Who is the study for?
This trial is for young patients (0-21 years at diagnosis) with medulloblastoma, a type of brain cancer. They must have specific molecular risk factors identified and be in stable condition post-treatment. Participants need to have good organ function, acceptable blood counts, and agree to use birth control if applicable. Those who are pregnant or breastfeeding cannot join.
What is being tested?
The study tests Difluoromethylornithine (DFMO) as a maintenance therapy for high-risk or relapsed medulloblastoma in children and young adults. It's an open-label trial, meaning both doctors and participants know what treatment is being given.
What are the potential side effects?
While the side effects of DFMO in this specific setting aren't detailed here, common ones may include stomach upset, hearing changes, mood swings, dizziness or sleepiness. Each patient's experience can vary.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My cancer is a specific type without certain genetic changes.
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My cancer is high-risk due to specific genetic features.
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I was diagnosed with my condition before turning 22.
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I can do most activities and my doctor thinks I have at least 2 months to live.
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My medulloblastoma's type was confirmed with specific genetic tests.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am 5 or older and my cancer has returned or didn't respond to treatment, and I haven't had radiation.
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I am not currently on any cancer treatments and have recovered from previous ones.
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My cancer has spread outside of my brain and spinal cord.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years plus 5 years follow up
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years plus 5 years follow up for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of participants with event free survival (EFS) during study
Secondary study objectives
Determine amount of DFMO in the CSF at 3 hours post dose
Determine the Overall Response Rate (ORR) of Participants using Modified RANO Criteria
Length of time that participants experience Overall Survival (OS)
+1 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Difluoromethylornithine (DFMO)Experimental Treatment1 Intervention
study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 2500 mg/m2 BID on each day of study.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Difluoromethylornithine
2015
Completed Phase 1
~50

Find a Location

Who is running the clinical trial?

Giselle ShollerLead Sponsor
22 Previous Clinical Trials
2,372 Total Patients Enrolled
6 Trials studying Medulloblastoma
376 Patients Enrolled for Medulloblastoma
Giselle SaulnierShollerLead Sponsor
21 Previous Clinical Trials
2,287 Total Patients Enrolled
6 Trials studying Medulloblastoma
376 Patients Enrolled for Medulloblastoma
Wake Forest University Health SciencesLead Sponsor
1,395 Previous Clinical Trials
2,460,087 Total Patients Enrolled
7 Trials studying Medulloblastoma
385 Patients Enrolled for Medulloblastoma

Media Library

Difluoromethylornithine Clinical Trial Eligibility Overview. Trial Name: NCT04696029 — Phase 2
Medulloblastoma Research Study Groups: Difluoromethylornithine (DFMO)
Medulloblastoma Clinical Trial 2023: Difluoromethylornithine Highlights & Side Effects. Trial Name: NCT04696029 — Phase 2
Difluoromethylornithine 2023 Treatment Timeline for Medical Study. Trial Name: NCT04696029 — Phase 2
~55 spots leftby Mar 2028
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