Personalized Risk-Adapted Therapy in Post-Pubertal Patients With Newly-Diagnosed Medulloblastoma
Status:
Not yet recruiting
Trial end date:
2028-12-01
Target enrollment:
Participant gender:
Summary
Medulloblastoma is a rare brain malignancy, mainly affecting children. Treatment of this
rapidly growing tumor begins with maximal surgical removal plus radiation and chemotherapy.
Treatment toxicity is high. Post-pubertal and pediatric medulloblastomas are biologically and
prognostically different, which mandates age-adapted treatment strategies. Patients after
puberty bear an intermediate to high prognostic risk. This means that a large number of these
patients, are faced with death and/or disability (mainly neurocognitive). Therefore, the
scientific and medical need is high. One of the genetic subgroups of medulloblastoma, the
SHH-subgroup (Sonic HedgeHog- subgroup), is highly overrepresented in medulloblastoma
patients after puberty. This subgroup can be treated with a targeted therapy. The
investigators will therefore randomize patients and treat SHH-subgroup patients with
sonidegib and a reduction of radiotherapy dose in the experimental arm of the trial. The
hypothesis that this personalized risk-adapted therapy will improve outcomes in view of
increased efficacy and decreased toxicity.
Phase:
Phase 2
Details
Lead Sponsor:
European Organisation for Research and Treatment of Cancer - EORTC