Phase 2 Study of Oral Tovorafenib for Children and Young Adults With RAF-Altered Relapsed or Progressive Low-Grade Glioma and Advanced Solid Tumors

What this Trial Is About

Researchers are evaluating the safety and effectiveness of tovorafenib, an oral Type II RAF kinase inhibitor, in pediatric and young adult participants with relapsed or progressive low-grade glioma or advanced solid tumors. This Phase 2, open-label, multicenter study focuses on participants aged from 6 months to 25 years who have specific RAF-related genetic alterations confirmed through molecular tests. The study includes several phases screening, treatment, long-term extension, end of treatment visits, safety follow-up, and long-term assessments. Participants receive tovorafenib orally at a dose of 420 mgm weekly, adjusted for their body surface area according to dose rounding guidelines. The study includes three groups two for those with low-grade glioma including an expanded access group and one for those with advanced solid tumors. Treatment continues through the designated periods, with the option for long-term extension based on individual response and tolerability. Throughout the study, participants will undergo regular assessments including measurement of tumor response by imaging criteria RANO or RECIST v1.1, monitoring for adverse events, and evaluation of clinical chemistry and hematology parameters. Additional evaluations include electrocardiograms and visual acuity tests. Researchers will measure overall response rates and other outcomes over up to 48 months, with long-term safety and efficacy monitored through follow-up visits and extension phases.

A Study to Evaluate Tovorafenib in Pediatric and Young Adult Participants With Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors