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BCC016: DFMO for Medullo

This is a study of the drug DFMO (difluoromethylornithine) for medulloblastoma that has returned or not responded to treatment. DFMO is an oral drug that inhibits a certain enzyme (protein) in blood which is associated with a bad outcome in neuroblastoma cases. Cancer cells have pathways that drive the cancer to grow and DFMO targets the specific pathway of this enzyme to turn these cells off.

You will have exams, tests, and procedures while on the study to evaluate whether you can participate in the study and how you are doing while on the study. These include physical exams, blood tests, urine tests, bone marrow aspirate and biopsies, heart tests, hearing tests, and imaging evaluations such as MRI of your brain and spine. You will receive treatment on this study for a total of about 2 years. After treatment, you will have follow-up examinations and medical tests. We would like to continue to find out about your health for about 5 years after you complete the study.

Yes
 

Valerie Brown
Suzanne Treadway - at streadway@pennstatehealth.psu.edu or 717-531-3097
Pediatrics: Hematology/Oncology (HERSHEY)
 

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This study is NOT accepting healthy volunteers
NCT04696029
SITE00000984
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Inclusion Criteria:
You have had medulloblastoma that was either high risk or had relapsed or had not responded to therapy
You are 21 years old or younger
You must have no evidence of disease at this time

Exclusion Criteria:
You are currently taking another study drug or an anticancer drug
Cancer
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See this study on ClinicalTrials.gov
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Location Contacts
Hershey, PA ,