Sabine Mueller, MD, PhD
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.
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…
Inclusion Criteria: Newly Diagnosed Participants: * Newly diagnosed craniopharyngioma, as based on imaging characteristics and central radiology review. Participants will initially be screened within confines of a screening consent and only those participants with findings consistent with craniopharyngioma and without findings suggesting an indeterminate lesion or lesion of an alternative diagnosis (including abnormal tumor markers found in blood or cerebral spinal fluid (CSF), if completed as part of standard of care (SOC) work-up or if lesion concerning for alternate diagnosis) will move a…
Given orally