Tovorafenib Treatment for Children and Young Adults with Newly Diagnosed or Recurrent Craniopharyngioma

What this Trial Is About

Researchers are evaluating the tolerability and effectiveness of tovorafenib, a pan-RAF-kinase inhibitor, as a single treatment for children and young adults with newly diagnosed or recurrent craniopharyngioma. This phase 2 study aims to determine progression-free survival and maintenance of quality of life over 12 months, comparing results with historical data. The study also explores visual and neuroendocrine function over several years and investigates changes in tumor biology and patient satisfaction during therapy. Participants are divided into two groups those with newly diagnosed craniopharyngioma and those with recurrent disease. All participants undergo biopsy or resection as standard care. Before surgery, patients receive one dose of tovorafenib. Those with measurable disease after surgery continue weekly maintenance doses. Those with complete tumor removal enter a follow-up phase. Non-surgical recurrent cases also receive weekly tovorafenib. Treatment is oral and given at a recommended phase 2 dose. Throughout the study, participants complete physical function and quality of life assessments, as well as visual and neuroendocrine evaluations at set intervals up to five years. Tissue and fluid samples collected before and after treatment help researchers study tumor changes at molecular levels. Patient-reported outcomes assess satisfaction and therapy-related side effects in context of social and racial factors. Safety is monitored continuously, and participation may last several years depending on follow-up needs.

Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults