PNOC029_Tovorafenib for the treatment of newly diagnosed or recurrent craniopharyngioma in children and young adults

Condition studied

craniopharyngioma

Investigational medicinal product(s)

Eligibility

Primary endpoint

PFS12 (progression free survival at 12 months), QOL12 (quality of life at 12 months)

Endpoint detail

Visual Responsive ≥ 0.2 logMAR improvement in VA compared to baseline, Stable VA = does not meet criteria for Improved or Worsening VA compared to baseline, Visual Progressive Disease ≥ 0.2 logMAR worsening compared to baseline, Visual field assessments with Humphrey visual field test, Presence or absence of new from baseline: hypothalamic obesity, diabetes insipidus, growth hormone deficiency, adrenal insufficiency