Actively Recruiting
Phase II Trial of Eflornithine (DFMO) as Maintenance Therapy for Molecular High Risk, Very High Risk, and Relapsed/Refractory Medulloblastoma
Led by Giselle Sholler · Updated on 2026-04-28
118
Participants Needed
24
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the use of difluoromethylornithine (DFMO) as maintenance therapy for children and young adults with molecular high risk, very high risk, or relapsed/refractory medulloblastoma. This phase II, open-label, multicenter study aims to understand how this oral drug may help prevent cancer progression in these specific groups of medulloblastoma patients. Participants will receive oral DFMO at a dose of 2500 mg/m2 twice daily for 730 days (approximately 2 years). The study includes three groups: molecular high risk medulloblastoma, molecular very high risk medulloblastoma, and relapsed/refractory medulloblastoma. A total of 118 participants will be enrolled to ensure at least 107 evaluable subjects across all groups. During the study, participants will undergo evaluations including brain and spine MRI scans, tumor assessments, and laboratory tests to monitor safety and drug levels. Researchers will measure event-free survival over 2 years plus 5 years of follow-up, overall survival up to 7 years, and record any adverse events. The study also tracks the drug concentration in cerebrospinal fluid. Participants are monitored closely throughout the treatment and follow-up periods, which may last several years.
CONDITIONS
Brief Title
DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 0 to 21 years at diagnosis
- Pathologically confirmed medulloblastoma with molecular grouping identified by Nanostring or methylation profiling
- Cohort 1: Metastatic non-MYC amplified Group 3, Metastatic Group 4, or Metastatic non-WNT/non-SHH (non-MYC amplified)
- Cohort 2: Metastatic OR MYCN amplified OR TP53 mutant non-infant (>3 years) SHH, MYC amplified Group 3, or Non-WNT, non-SHH infant (<3 years)
- Cohort 3: Relapsed or refractory medulloblastoma
- Pre-enrollment tumor assessment including brain and spine MRI, lumbar puncture if previously positive, bone marrow biopsy if previously positive
- No evidence of disease or stable residual nonbulky disease (non-progression over 2 imaging studies at least 6 weeks apart; maximal cross-sectional area <3cm^2)
- Enrollment no later than 60 days after last conventional chemotherapy dose; at least 45 days after last stem cell transplant if applicable
- Lansky or Karnofsky Performance Scale score ≥50% and life expectancy ≥2 months
- Organ function tests within 7 days prior to first dose showing adequate hematological, liver, and renal function
- Negative pregnancy test for females of childbearing potential; agreement to use birth control and stop breastfeeding if lactating
- Written informed consent obtained
You will not qualify if you...
- Body surface area (BSA) less than 0.25 m2
- Metastatic disease outside the central nervous system
- Relapsed/refractory patients who are radiation-naïve and 5 years or older at enrollment
- Currently receiving another investigational drug
- Currently receiving other anticancer agents or not recovered from bone marrow suppression
- Uncontrolled infection until well controlled
- Inability to comply with safety monitoring requirements or expected poor compliance
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 730 days
Participants receive oral difluoromethylornithine (DFMO) twice daily as maintenance therapy.
Regular visits for monitoring as per study schedule
Duration - Up to 5 years after treatment
Participants are monitored for event-free survival, overall survival, and safety after treatment ends.
Periodic visits for outcome assessments
Trial Site Locations
Total: 24 locations
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
Actively Recruiting
3
Rady Children's Hospital
San Diego, California, United States, 92123
Actively Recruiting
4
Stanford University
Stanford, California, United States, 94305
Actively Recruiting
5
Connecticut Children's Hospital
Hartford, Connecticut, United States, 06106
Actively Recruiting
6
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
7
Arnold Palmer Hospital for Children
Orlando, Florida, United States, 32806
Actively Recruiting
8
All Children's Hospital Johns Hopkins Medicine
St. Petersburg, Florida, United States, 33701
Actively Recruiting
9
St. Joseph's Children's Hospital
Tampa, Florida, United States, 33607
Actively Recruiting
10
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States, 96813
Actively Recruiting
11
Advocate Aurora Research Institute
Chicago, Illinois, United States, 60453
Actively Recruiting
12
Kentucky Children's Hospital
Lexington, Kentucky, United States, 40502
Actively Recruiting
13
Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine
Louisville, Kentucky, United States, 40202
Actively Recruiting
14
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States, 64108
Actively Recruiting
15
Cardinal Glennon Children's Medical Center
St Louis, Missouri, United States, 63104
Actively Recruiting
16
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
Actively Recruiting
17
Levine Children's Hospital
Charlotte, North Carolina, United States, 28204
Actively Recruiting
18
University of Oklahoma
Oklahoma City, Oklahoma, United States, 73104
Actively Recruiting
19
Randall Children's Hospital
Portland, Oregon, United States, 97227
Actively Recruiting
20
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
21
Hasbro Children's Hospital
Providence, Rhode Island, United States, 02903
Actively Recruiting
22
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Actively Recruiting
23
Dell Children's Blood and Cancer Center
Austin, Texas, United States, 78723
Actively Recruiting
24
CHUQ
Québec, Quebec, Canada, QC G1V 4W6
Actively Recruiting
Research Team
B
BCC Enroll
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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