Medulloblastoma is a rare brain cancer that predominantly affects children. However, it affects approximately 550 adults in the United States and 450 adults in Europe annually, with the majority being young adults. Treatment consists of surgical removal, radiation therapy, and maintenance chemotherapy. Treatment toxicity is high and often results in decline of cognition, fertility, neurological function and hearing, as well as severe impairments of quality of life, social, and professional function. Adolescent and adult medulloblastomas are biologically distinct from pediatric medulloblastomas, which mandates age-adapted treatment strategies. Adolescents and adults have a high incidence of SHH-mutated tumors; therefore, the investigators will utilize sonidegib, which targets these mutations. This study will assess if the addition of sonidegib can increase the length of time before the cancer spreads or grows in post-pubertal patients with newly diagnosed SHH-activated medulloblastoma compared to standard of care alone. This is the first trial worldwide that prospectively enrolls post-pubertal patients with medulloblastoma in a randomized comparative phase II trial.
Trial Registration: ClinicalTrials.gov Identifier: NCT04402073