Phase I Trial Evaluating Safety and Effects of Tovorafenib in Japanese Children and Young Adults with BRAF-altered Recurrent or Progressive Low-grade Glioma

What this Trial Is About

Researchers are evaluating the safety and how the body processes the drug tovorafenib in Japanese children, adolescents, and young adults with specific brain tumors called low-grade gliomas that have BRAF gene alterations. This study also aims to assess how well tovorafenib works to shrink these brain tumors. Participants must be between 6 months and 25 years old and have relapsed or progressive tumors confirmed by molecular testing and imaging. All participants receive tovorafenib orally once a week. The study includes four periods a screening period lasting up to 4 weeks to determine eligibility a treatment period lasting up to 24 months where participants take tovorafenib with in-person visits especially frequent during the first two months and monthly thereafter a 30-day safety follow-up after treatment ends and a long-term follow-up for up to 2 years with visits every 3 months unless new treatment starts or they leave the study. Treatment continues as long as the disease does not worsen and the drug is tolerated. Participants will attend several clinic visits, provide blood and urine samples, and undergo health assessments during the study, which may last up to about 4 years. Researchers will monitor side effects, measure drug levels in the blood over time, and track tumor size every 12 weeks. Safety is followed closely during and after treatment, and participants may switch to commercially available tovorafenib once approved in Japan. Participants may leave the study at any time without penalty.

A Study to Assess a Medicine Called Tovorafenib in Japanese Children and Young Adults With Brain Tumours