MASTER KEY Project: Powering Clinical Development for Rare Cancers Through a Platform Trial

Area of Science:

• Oncology
• Genomics
• Clinical Trials

Background:

• Rare cancers present significant therapeutic challenges compared to common malignancies.
• Establishing standard treatment protocols for rare cancers is complex and requires innovative approaches.
• The MASTER KEY Project addresses the unmet need for effective therapies in rare and unknown primary cancers.

Purpose of the Study:

• To establish a multicenter prospective registry and conduct multiple clinical trials for rare cancers.
• To accumulate high-quality data for future drug development and therapeutic strategy refinement.
• To investigate the prevalence of genetic abnormalities and evaluate treatment efficacy in rare cancer patients.

Main Methods:

• Prospective registry accumulating consecutive patient data.
• Simultaneous multiple clinical trials targeting specific biomarkers or rare tumor types.
• Next-generation sequencing (NGS) to identify actionable genetic alterations.

Main Results:

• 560 patients enrolled between May 2017 and April 2019.
• 48% of patients tested with NGS had actionable genetic alterations.
• 13% of patients were enrolled in clinical trials, with an accrual rate of 3.94 patients/month.

Conclusions:

• The MASTER KEY Project demonstrates the value of a comprehensive platform trial strategy for rare cancers.
• Identifying actionable alterations through comprehensive genomic profiling is feasible in rare cancer populations.
• This initiative is expected to accelerate the development of novel treatments for rare and difficult-to-treat cancers.