A phase 1/2, open-label, safety, tolerability, pharmacokinetics, and anti-tumor activity study of repotrectinib in pediatric and young adult subjects with advanced or metastatic malignancies harboring alk, ros1, or ntrk1-3 alterations

A single drug, repotrectinib, is being tested in children and young adults with advanced cancers that have specific genetic faults in the ALK, ROS1, or NTRK1-3 genes. These genetic alterations drive tumour growth but are rare in paediatric cancers, meaning few treatment options exist. Standard chemotherapy often fails or causes severe side effects in these patients. Repotrectinib is a targeted therapy designed to block the faulty proteins produced by these genes, potentially shrinking tumours or halting their progression. The current study is a phase 1/2 trial to confirm the safest and most effective dose, and to measure how the drug behaves in young bodies. If successful, this research could provide a new, less toxic treatment option for children and young adults with these specific hard-to-treat cancers. It would fill a gap where no targeted therapies are currently approved for this age group. The impact would be directly on patients and families, offering a chance for better outcomes without the harsh side effects of conventional chemotherapy. This is a practical, patient-focused trial aimed at bringing a precision medicine to a small but underserved population.