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MASTER KEY Project: Powering Clinical Development for Rare Cancers Through a Platform Trial
Hitomi S Okuma1, Kan Yonemori2, Shoko N Narita3
1Department of Breast and Medical Oncology, Clinical Research Support Office, National Cancer Center Hospital, Tokyo, Japan.
Abstract:
For rare cancers, challenges in establishing standard therapies are greater than those for major cancers, and effective methods are needed. MASTER KEY Project is a multicenter study based in Japan, with two main parts: prospective registry study and multiple clinical trials. Advanced rare cancers, cancers of unknown primary origin, and those with rare tissue subtypes of common cancers are targeted. The registry study accumulates highly reliable consecutive data that can be used for future drug development. The multiple trials are conducted simultaneously, targeting either a specific biomarker or a rare tumor type of interest. The first interim data set from the registry part presented here shows the prevalence of genetic abnormalities, response rates, survival rates, and clinical trial enrollment rates. From May 2017 to April 2019, 560 patients (mean age = 53) were enrolled in the project. Frequent cancer types included soft tissue sarcomas, neuroendocrine tumors, and central nervous system tumors. Among the 528 patients with assessable data, 69% (364/528) had next-generation sequencing tests, with 48% (176/364) harboring an "actionable" alteration. Seventy-one (13%) patients have been enrolled in one of the clinical trials, with an accrual rate of 3.94 patients/month. A descriptive analysis of biomarker-directed or non-biomarker-directed treatment survival was performed. This project is expected to accelerate development of treatments for rare cancers and show that comprehensive platform trials are an advantageous strategy.
Insights
The MASTER KEY Project, a Japanese study, is accelerating rare cancer treatment development. It uses a registry and clinical trials to identify actionable genetic alterations and improve patient outcomes for rare and unknown primary cancers.
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.
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