PNOC029 is a clinical trial testing tovorafenib in children and young adults with newly diagnosed or recurrent craniopharyngioma, a rare brain tumour that can affect vision, hormones, growth, and quality of life.
The study is looking at whether tovorafenib can help control tumour growth and preserve quality of life. Participants may receive tovorafenib before planned biopsy or surgery, and some may continue weekly treatment afterwards for up to 24 months if appropriate. The study also monitors safety, tumour response, vision, hormone function, and quality of life over time.
PNOC029 is a Phase II, open-label study of tovorafenib for patients aged 1 to 39 years with newly diagnosed or recurrent craniopharyngioma. Participants receive a pre-operative dose of tovorafenib before standard-of-care biopsy or resection. Those with residual or measurable disease may continue once-weekly oral tovorafenib in 28-day cycles for up to 24 months, unless there is disease progression or unacceptable toxicity. The primary aim is to assess 12-month progression-free survival and maintenance of physical quality of life compared with historical controls. Secondary assessments include safety, radiographic response, visual outcomes, neuroendocrine outcomes, and longer-term quality of life.