BCC 016 A Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma

Objectives and Summary

Medulloblastoma is the most common pediatric malignant brain tumor. While advances in therapy have resulted in improved outcomes for standard risk medulloblastoma, outcomes in high-risk and relapsed medulloblastoma have continued to be inferior. As such, research communities are investigating alternative treatment regimens that may improve outcomes with minimal added toxicity for patients with high-risk/very high-risk medulloblastoma, including for patients with relapsed disease. This study seeks to evaluate how well the enzyme inhibitor difluoromethylornithine (DFMO) works to prevent relapse in patients with high risk and very high risk medulloblastoma who have completed conventional (first line) therapy, as well as patients with medulloblastoma who have relapsed and received one line of salvage therapy and are in remission. This treatment will be given as a maintenance treatment after therapy to assess if fewer patients relapse.

Minimal criteria in order to be included in this study:

• Age 0-21 at time of diagnosis of medulloblastoma
• A pathologically confirmed diagnosis of medulloblastoma
• Must have no evidence of disease on scans or minimal disease that has been stable
• MRI of the brain and spine
• Have received conventional therapy within the previous 60 days or autologous stem cell transplant within the previous 120 days
• Clinically adequate organ function
• Must not be pregnant or breastfeeding
• Must not have metastatic disease outside of the central nervous system
• Must not be receiving other investigational medications or anti-cancer agents at the time of enrollment
• Must not have active, uncontrolled infections

Clinicaltrials.gov Study Details >