← Back to Search

DFMO for Medulloblastoma

Q: Are there any significant risks associated with utilizing Difluoromethylornithine therapeutically?

Our team has estimated that the safety rating of Difluoromethylornithine to be a 2, as only limited data exists in support of its safety without evidence for efficacy.

Q: Are there any precedents to this research involving Difluoromethylornithine?

Currently, 11 clinical trials are underway for Difluoromethylornithine with 2 of them in the last stage. While most studies occur in Boise <a href="https://www.withpower.com/clinical-trials/idaho">Idaho</a>, there is a global presence spanning 1381 sites around the world.

Q: Does this research represent a novel approach to the topic?

Since 2013, Difluoromethylornithine has been under investigation. The initial trial was conducted by <a href="https://www.withpower.com/clinical-trials/cancer">Cancer</a> Prevention Pharmaceuticals and included 1340 participants. Upon successful Phase 3 approval of the drug in that same year, 11 more studies have since commenced across 699 cities and 12 nations.

Q: How many participants are undertaking the research?

The study requires 118 qualified candidates for participation. These individuals can join from St. Joseph's Children's Hospital in Tampa, <a href="https://www.withpower.com/clinical-trials/kentucky">Kentucky</a> and Norton Children's Hospital/University of Louisville in Louisville, <a href="https://www.withpower.com/clinical-trials/missouri">Missouri</a>.

Q: Are there any unfilled slots for people to join this research?

Indeed, the data presented on clinicaltrials.gov implies that this research is actively enrolling participants as of now. The experiment was first put forward on March 29th 2021 and recently revised August 3rd 2022; 118 patients are being recruited from 13 distinct sites.

Q: Are there any North American sites participating in this experimentation?

Thirteen healthcare institutions are currently partaking in this investigation, such as St. Joseph's Children's Hospital in Tampa, Norton Children's Hospital/University of Louisville in Louisville, and Cardinal Glennon Children’s Medical Center located within Saint Louis - to name a few.

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)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years plus 5 years follow up

Awards & highlights

Study 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.Check my eligibility

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.See study design

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

Select...

My cancer is a specific type without certain genetic changes.

Select...

My cancer is high-risk due to specific genetic features.

Select...

I was diagnosed with my condition before turning 22.

Select...

I can do most activities and my doctor thinks I have at least 2 months to live.

Select...

My medulloblastoma's type was confirmed with specific genetic tests.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years plus 5 years follow up

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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

Secondary outcome measures

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

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

0 / 6Eligibility criteria met

Find a Location

Who is running the clinical trial?

Giselle ShollerLead Sponsor

21 Previous Clinical Trials

2,087 Total Patients Enrolled

6 Trials studying Medulloblastoma

372 Patients Enrolled for Medulloblastoma

Giselle SaulnierShollerLead Sponsor

21 Previous Clinical Trials

2,100 Total Patients Enrolled

6 Trials studying Medulloblastoma

372 Patients Enrolled for Medulloblastoma

Wake Forest University Health SciencesLead Sponsor

1,241 Previous Clinical Trials

1,004,119 Total Patients Enrolled

7 Trials studying Medulloblastoma

381 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any significant risks associated with utilizing Difluoromethylornithine therapeutically?

"Our team has estimated that the safety rating of Difluoromethylornithine to be a 2, as only limited data exists in support of its safety without evidence for efficacy."

Answered by AI

Are there any precedents to this research involving Difluoromethylornithine?

"Currently, 11 clinical trials are underway for Difluoromethylornithine with 2 of them in the last stage. While most studies occur in Boise Idaho, there is a global presence spanning 1381 sites around the world."

Answered by AI

Does this research represent a novel approach to the topic?

"Since 2013, Difluoromethylornithine has been under investigation. The initial trial was conducted by Cancer Prevention Pharmaceuticals and included 1340 participants. Upon successful Phase 3 approval of the drug in that same year, 11 more studies have since commenced across 699 cities and 12 nations."

Answered by AI

How many participants are undertaking the research?

"The study requires 118 qualified candidates for participation. These individuals can join from St. Joseph's Children's Hospital in Tampa, Kentucky and Norton Children's Hospital/University of Louisville in Louisville, Missouri."

Answered by AI

Are there any unfilled slots for people to join this research?

"Indeed, the data presented on clinicaltrials.gov implies that this research is actively enrolling participants as of now. The experiment was first put forward on March 29th 2021 and recently revised August 3rd 2022; 118 patients are being recruited from 13 distinct sites."

Answered by AI

Are there any North American sites participating in this experimentation?

"Thirteen healthcare institutions are currently partaking in this investigation, such as St. Joseph's Children's Hospital in Tampa, Norton Children's Hospital/University of Louisville in Louisville, and Cardinal Glennon Children’s Medical Center located within Saint Louis - to name a few."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

Giselle Sholler 2021. "DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04696029.

All > Medulloblastoma