Tovorafenib for the Treatment of Newly Diagnosed or Recurrent Craniopharyngioma in Children and Young Adults
Sabine Mueller, MD, PhD
Summary
The current study assesses the tolerability and efficacy of monotherapy with pan-RAF-kinase (Tovorafenib) inhibition for the treatment of children and young adults with craniopharyngioma.
Description
PRIMARY OBJECTIVE: I. To determine progression free survival and maintenance of quality of life at 12 months as based on physical function and compared to historical controls. SECONDARY OBJECTIVES: I. To identify proportion of participants with visual deficits at 1-year, 2-year, and 3-year follow-up. II. To identify proportion of participants with neuroendocrine deficits at 1-year, 2-year, and 3-year follow-up. EXPLORATORY OBJECTIVES: I. To assess Quality of Life (QOL) and cognitive measures in children and young adults with newly diagnosed or recurrent craniopharyngioma. II. To perform…