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

Recruiting now · Phase 2

Conditions studied: Medulloblastoma

In brief

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.

Key facts

Study ID

NCT04696029

Run by

Giselle Sholler

People needed

118

Starts

2021-03-29

Expected to finish

2029-03-01

Last updated by the study team

2026-04-28

Who can join

Age: any, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age: 0-21 years of age at diagnosis
• Pathology All patients must either have a pathologically confirmed diagnosis of medulloblastoma with molecular grouping identified by either Nanostring or methylation profiling.
• Cohort 1- Molecular High Risk:
• Metastatic non-MYC amplified Group 3
• Metastatic Group 4
• Metastatic non-WNT/non-SHH (Must be non-MYC amplified)
• Cohort 2- Molecular Very High Risk
• Metastatic OR MYCN amplified OR TP53 mutant non-infant (>3 yrs) SHH
• MYC amplified Group 3
• Non-WNT, non-SHH infant (< 3 yrs)
• Cohort 3: Relapsed/Refractory Medulloblastoma
• Pre-enrollment tumor survey:
• Prior to enrollment on this study, a determination of mandatory disease staging must be performed:
• Tumor imaging studies including: Brain and spine MRI
• Lumbar Puncture only if previously positive
• Bone Marrow aspiration/biopsy only if previously positive
• This disease assessment is required for eligibility and preferably should be done within 2 weeks prior to first dose of study drug, but must be done within a maximum of 4 weeks before first dose of study drug.
• Disease Status: Subjects must have no evidence of disease, or stable* residual nonbulky** disease.
• Stable residual disease defined as non-progression over 2 separate imaging studies at least 6 weeks apart
• *Non-bulky disease defined as maximal cross-sectional area < 3cm\^2 at enrollment. Patients with leptomeningeal disease are allowed to participate on study.
• Timing from prior therapy:
• Enrollment (first dose of DFMO) no later than 60 days after last dose of conventional chemotherapy. Patients who have undergone high dose chemotherapy (HDCT) with autologous stem cell transplantation (SCT) are eligible if more than 45 days have elapsed since date of last SCT.
• Patients must have a Lansky or Karnofsky Performance Scale score of ≥ 50% (see Appendix II) and patients must have a life expectancy of ≥ 2 months.
• All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to first dose of study drug unless otherwise indicated below.
• Patients must have adequate organ functions at the time of registration:

You may not qualify if…

• BSA of <0.25 m2
• Metastatic disease outside of CNS
• Relapsed/refractory patients who are radiation-naïve and age 5 years or older at time of enrollment
• Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
• 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.
• Infection: 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, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States (enrolling)
• UCSF Benioff Children's Hospital Oakland — Oakland, California, United States (enrolling)
• Rady Children's Hospital — San Diego, California, United States (enrolling)
• Stanford University — Stanford, California, United States (enrolling)
• Connecticut Children's Hospital — Hartford, Connecticut, United States (enrolling)
• Nicklaus Children's Hospital — Miami, Florida, United States (enrolling)
• Arnold Palmer Hospital for Children — Orlando, Florida, United States (enrolling)
• All Children's Hospital Johns Hopkins Medicine — St. Petersburg, Florida, United States (enrolling)
• St. Joseph's Children's Hospital — Tampa, Florida, United States (enrolling)
• Kapiolani Medical Center for Women and Children — Honolulu, Hawaii, United States (enrolling)
• Advocate Aurora Research Institute — Chicago, Illinois, United States (enrolling)
• Kentucky Children's Hospital — Lexington, Kentucky, United States (enrolling)
• Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine — Louisville, Kentucky, United States (enrolling)
• Children's Mercy Hospitals and Clinics — Kansas City, Missouri, United States (enrolling)
• Cardinal Glennon Children's Medical Center — St Louis, Missouri, United States (enrolling)
• Hackensack University Medical Center — Hackensack, New Jersey, United States (enrolling)
• Levine Children's Hospital — Charlotte, North Carolina, United States (enrolling)
• University of Oklahoma — Oklahoma City, Oklahoma, United States (enrolling)
• Randall Children's Hospital — Portland, Oregon, United States (enrolling)
• Penn State Milton S. Hershey Medical Center and Children's Hospital — Hershey, Pennsylvania, United States (enrolling)
• Hasbro Children's Hospital — Providence, Rhode Island, United States (enrolling)
• Medical University of South Carolina — Charleston, South Carolina, United States (enrolling)
• Dell Children's Blood and Cancer Center — Austin, Texas, United States (enrolling)
• CHUQ — Québec, Quebec, Canada (enrolling)

Full record on ClinicalTrials.gov

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