Development of Randomized Trials in Adults with Medulloblastoma—The Example of EORTC 1634-BTG/NOA-23

Medulloblastoma is a rare brain malignancy. Patients after puberty are and bear an intermediate prognosis. Standard treatment consists of maximal resection plus radio-chemotherapy. Treatment toxicity high produces disabling long-term side effects. The sonic hedgehog (SHH) subgroup highly overrepresented in the post-pubertal adult population can be targeted by smoothened (SMO) inhibitors. No practice-changing prospective randomized data have been generated adults. EORTC 1634-BTG/NOA-23 trial will randomize patients between standard-dose vs. reduced-dosed craniospinal radiotherapy SHH-subgroup SMO inhibitor sonidegib (OdomzoTM, Sun Pharmaceuticals Industries, Inc., New York, USA) addition to standard radio-chemotherapy alone improve outcomes view decreased radiotherapy-related increased efficacy. We further investigate tumor tissue, blood, cerebrospinal fluid as well magnetic resonance imaging plans generate information that helps outcomes. Given effects typically occur late, follow-up monitor classic therapy, but also health-related quality life, cognition, social professional outcome, reproduction fertility. In summary, we unprecedented translated into changes with medulloblastoma help design future clinical trials.