Study of Repotrectinib for Children and Young Adults With Advanced Cancers Having ALK, ROS1, or NTRK1-3 Gene Changes Evaluating Safety, Tolerance, and Effectiveness in Phase 1 and 2

What this Trial Is About

Researchers are evaluating repotrectinib, an oral drug, in pediatric and young adult patients with advanced or metastatic cancers that have specific genetic changes in ALK, ROS1, or NTRK1-3 genes. The study includes two phases Phase 1 focuses on finding safe dose levels and the recommended dose for children under 12, while Phase 2 assesses the drugs anti-tumor activity in patients aged 12 to 25 with certain gene fusions or alterations. This study aims to address cancers that have progressed despite other treatments or have no standard cure options. In Phase 1, about 12 children under 12 years old with advanced solid tumors or lymphoma will receive different doses of repotrectinib to determine safety and the best dosing. Phase 2 includes three groups of patients aged 12 to 25, with varying genetic profiles and prior treatment histories, all receiving oral repotrectinib. The drug will be given continuously in cycles, with careful monitoring. Enrollment for Phase 1 and Phase 2 occurs simultaneously, allowing younger children to join Phase 2 once their dose is established. Participants will undergo regular assessments including scans and tests to measure tumor response, drug levels in the blood, and side effects. The study tracks several outcomes such as response rate, duration of response, survival, and brain tumor progression over approximately three years. Safety and tolerability are closely monitored during the first 28 days of treatment, with ongoing follow-up visits throughout the study period. This long-term observation helps researchers understand both the drugs effects and patient well-being.

A Study of Repotrectinib in Pediatric and Young Adult Subjects Harboring ALK, ROS1, OR NTRK1-3 Alterations