Precision Medicine for Rare Cancers

Precision Medicine for Rare Cancers

Precision medicine trial for rare cancers. Phase II histology-agnostic study using comprehensive genomic profiling for targeted therapy.

A Phase II Trial of Precision Medicine Approach in Rare Cancers

Full Title

A phase II, open-label, histology-agnostic study of molecularly-targeted therapy based on comprehensive genomic profiling in patients with advanced rare solid tumors

Purpose

Rare cancers often lack specific treatments, but many have genetic changes that can be targeted with existing drugs approved for other cancers. This study uses comprehensive genetic testing to match rare cancer patients with targeted drugs based on their tumor’s genetic makeup.

Primary Objectives

  • Evaluate overall response rate to genomically-matched targeted therapy
  • Assess feasibility of comprehensive genomic profiling-guided treatment selection
  • Determine progression-free survival and overall survival outcomes

Diseases

  • Sarcomas
  • Neuroendocrine Tumors
  • Bile Duct Cancer
  • Adrenal Cancer,
  • Thymic Tumors
  • Rare Solid Tumors

Who Can Join this Trial

To participate in this study, you must meet ALL of the following criteria

  • Have histologically confirmed advanced or metastatic rare solid tumor
  • Tumor must harbor at least one actionable genetic alteration identified by comprehensive genomic profiling
  • Have received at least one line of standard therapy OR no standard therapy exists
  • Have at least one measurable lesion per RECIST v1.1 criteria
  • ECOG performance status 0-2
  • Age 18 years or older
  • Life expectancy ≥12 weeks
  • Adequate organ function including blood counts, liver, and kidney function
  • Tissue available for comprehensive genomic profiling if not previously performed
  • Targeted therapy must be available for the identified genetic alteration