Molecular Targeted Maintenance Therapy Versus Standard of Care in Advanced Biliary Cancer: an International, Randomised, Controlled, Open-label, Platform Phase 3 Trial

What this Trial Is About

Researchers are investigating whether adding a targeted therapy after four cycles of the current standard treatment for advanced biliary cancer is better than continuing the standard treatment alone. This Phase 3, multicenter, randomized, open-label trial aims to identify patients whose tumors have specific molecular changes that can be targeted with new therapies. Patients first undergo a screening phase to get a detailed genetic profile of their tumor and gather baseline health and disease information. During the trial, patients who show disease control after four cycles of standard first-line therapy and have tumors with targetable molecular alterations will be randomly assigned in a 2:1 ratio to receive either a personalized targeted therapy matched to their tumor's genetics or to continue the standard treatment. The targeted therapies include various drugs such as Futibatinib, Ivosidenib, Zanidatamab, and combinations like Trastuzumab with Neratinib, among others. Drug dosing schedules vary by medication, with some given daily and others every few weeks. Participants will be closely monitored through regular assessments including blood and tumor sample collection, imaging, and evaluations of their overall health and tumor responses. The main measurement is progression-free survival, tracking the time from randomization until the disease worsens or death, for up to five years. Additional outcomes include overall survival, response rates, and tumor size changes. The study includes safety monitoring and requires participants to follow scheduled visits and procedures throughout the trial period, which may last several years.

Personalized Medicine for Advanced Biliary Cancer Patients