DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma
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.
| Condition(s) | Medulloblastoma |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| Summary | 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. |
| Who can participate | Inclusion Criteria: 1. Age: 0-21 years of age at diagnosis 2. 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 3. 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 Punc |
| Ages | 21 Years |
| Sex | All |
| Lead sponsor | Giselle Sholler |
| Locations | Little Rock, Arkansas, United States; Oakland, California, United States; San Diego, California, United States; Stanford, California, United States; Hartford, Connecticut, United States; Miami, Florida, United States (+18 more sites) |
| Start date | 2021-03-29 |
| NCT ID | NCT04696029 |
| Official listing | https://clinicaltrials.gov/study/NCT04696029 |