Actively Recruiting
DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma
Led by Giselle Sholler · Updated on 2026-04-28
118
Participants Needed
24
Research Sites
413 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma.
CONDITIONS
Official 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
- For Cohort 1: Metastatic non-MYC amplified Group 3, Metastatic Group 4, or Metastatic non-WNT/non-SHH (non-MYC amplified)
- For 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)
- For Cohort 3: Relapsed or refractory medulloblastoma
- Tumor imaging studies (brain and spine MRI) within 4 weeks before first dose; lumbar puncture and bone marrow aspiration if previously positive
- No evidence of disease or stable residual non-bulky disease (max cross-sectional area <3 cm²)
- Enrollment no later than 60 days after last conventional chemotherapy dose; >45 days after autologous stem cell transplant if applicable
- Lansky or Karnofsky Performance Scale score ≥ 50% and life expectancy ≥ 2 months
- Adequate organ function within 7 days before first dose
- Negative pregnancy test for females of childbearing potential and agreement to effective birth control; lactating females must stop breast-feeding
- Written informed consent obtained
You will not qualify if you...
- Body surface area less than 0.25 m²
- Metastatic disease outside the central nervous system
- Relapsed/refractory patients who are radiation-naïve and aged 5 years or older
- Currently receiving another investigational drug
- Currently receiving other anticancer agents or not fully recovered from prior chemotherapy effects
- Uncontrolled infection
- Inability or likely inability to comply with study safety monitoring requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
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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