Platform Study of Genotyping Guided Precision Medicine for Rare Tumors in China

What this Trial Is About

Researchers are conducting a Phase II, open-label, non-randomized clinical trial at a single center to study patients with advanced rare solid tumors who have not responded to standard treatments. The study focuses on evaluating the safety and effectiveness of targeted drugs for tumors with specific genetic changes and immune checkpoint inhibitors for tumors without such changes. This trial addresses the lack of established guidelines for treating rare tumors and involves multiple treatment arms based on patients' tumor genetic profiles. Participants with certain gene alterations such as EGFR mutations, ALK gene fusion, ROS-1 fusion, and others will receive targeted therapies including drugs like Dacomitinib and Crizotinib. Those without these alterations will be treated with the immune checkpoint inhibitor Sintilimab. Additionally, patients who develop resistance to initial treatments may receive combination therapies involving drugs such as Niraparib with Sintilimab, or Vemurafenib with Atezolizumab. Treatment continues until disease progression or unacceptable side effects occur. During the study, patients will undergo regular assessments including imaging to measure tumor response, evaluations for side effects, and monitoring of overall survival and progression-free survival for up to two years. The primary outcome is the objective response rate, determined by independent and investigator reviews. Secondary outcomes include progression-free survival, duration of response, disease control rate, and incidence of adverse events. Participants will provide tissue samples for molecular typing and follow clinical and follow-up plans throughout the trial.

PLATFORM Study of Precision Medicine for Rare Tumors