Phase 2 Study of Oral Difluoromethylornithine DFMO as Maintenance Treatment for Children and Young Adults With High Risk or Relapsed Medulloblastoma

What this Trial Is About

Researchers are evaluating the use of oral difluoromethylornithine DFMO as maintenance therapy for children and young adults with molecular high risk, very high risk, or relapsedrefractory medulloblastoma. This phase II, open-label, multicenter study aims to understand how DFMO affects event-free survival over two years plus five years of follow-up, along with other outcomes such as overall survival and safety. Participants will receive DFMO orally at a dose of 2500 mgm2 twice daily every day for 730 days approximately two years. The study includes three specific groups based on disease status molecular high risk medulloblastoma, molecular very high risk medulloblastoma, and relapsedrefractory medulloblastoma. Each cohort will enroll between 32 and 39 participants, with a total of 118 subjects planned for the study. During the study, participants will be closely monitored using brain and spine MRI imaging, lumbar puncture or bone marrow tests if previously positive, and assessments of blood and organ function. Researchers will track event-free survival, overall survival, response rates, adverse events, and DFMO levels in cerebrospinal fluid. The study includes follow-up for up to seven years to observe long-term outcomes and safety. Participants will be evaluated regularly throughout the two-year treatment and follow-up periods.

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