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Proffered Paper – Developmental therapeutics

441O - A Platform Trial with a Registry Study for Rare Cancers: MASTER KEY Project


29 Sep 2019


Proffered Paper – Developmental therapeutics


Rare Cancers

Tumour Site


Shoko Narita


Annals of Oncology (2019) 30 (suppl_5): v159-v193. 10.1093/annonc/mdz244


S.N. Narita1, H.S. Okuma1, K. Yonemori2, T. Sukigara1, A. Hirakawa3, W. Munakata4, N. Okita1, T. Shimizu5, T. Shibata6, A. Kawai7, N. Yamamoto5, K. Izutsu4, M. Muto8, H. Akita9, E. Baba10, K. Nakamura1, H. Mano11, T. Nishida12, Y. Fujiwara1

Author affiliations

  • 1 Clinical Research Support Office, National Cancer Center Hospital, 104-0045 - Tokyo/JP
  • 2 Department Of Breast And Medical Oncology, National Cancer Center Hospital, 104-0045 - Tokyo/JP
  • 3 Department Of Biostatistics And Bioinformatics, The University of Tokyo, 113-0033 - Tokyo/JP
  • 4 Department Of Hematology, National Cancer Center Hospital, 104-0045 - Tokyo/JP
  • 5 Department Of Experimental Therapeutics, National Cancer Center Hospital, 104-0045 - Tokyo/JP
  • 6 Center For Research Administration And Support Director, National Cancer Center, 1040045 - Tokyo/JP
  • 7 Department Of Musculoskeletal Oncology And Rehabilitation, National Cancer Center Hospital, 104-0045 - Tokyo/JP
  • 8 Department Of Clinical Oncology, Kyoto University Hospital, 606-8507 - Kyoto/JP
  • 9 Department Of Medical Oncology, Hokkaido University Hospital, 060-8648 - Sapporo/JP
  • 10 Department Of Hematology, Oncology & Cardiovascular Medicine, Kyushu University Hospital, 8128582 - Fukuoka/JP
  • 11 Division Of Cellular Signaling, National Cancer Center Research Institute, 104-0045 - Tokyo/JP
  • 12 Department Of Gastric Surgery, National Cancer Center Hospital, 104-0045 - Tokyo/JP


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Abstract 441O


Rare cancers have had a challenge in establishing standard therapies for patients compared to major cancers, due to the lack of basis for clinical studies and investigations. We started a biomarker driven basket/umbrella trial using a “master protocol”, called the MASTER KEY Project, which aims to find more efficient ways to evaluate treatments for rare cancers.


The project opened in April 2017 and consists of a prospective registry study part and a multiple clinical trials part. Patients with advanced rare cancers (annual incidence <6 / 100,000 population)/cancers of unknown primary/rare tissue subtypes of common cancers, undergoing a molecular diagnostic testing are enrolled. The primary objective of the registry study is to collect consecutive data on biomarker, patient background, and prognosis to build a database highly reliable for use as historical control data in future clinical trials. Sub-studies are placed under a “master protocol”, and each sub-study will enroll patients with the appropriate biomarker, regardless of tumor type.


As of Feb. 2019, 515 patients were enrolled in the project, and 493 were available for their backgrounds. Most frequent cancer types were: soft tissue sarcomas (33.7%), neuroendocrine tumors (7.3%), tumors of CNS (5.9%), salivary gland tumors (4.5%), etc. 346 patients had next generation sequencing testing results. The most common alterations were TP53 (32.4%), KRAS (10.4%), PIK3CA (9.5%), MDM2 (5.2%), CDK42 (4.9%), CDKN2A (4.9%), and RB1 (4.9%). 278 patients already had 6 months follow-up, and 116 patients received 181 systemic chemotherapy regimens after enrollment. Of those regimens, 25 were biomarker-based therapies, and median progression free survival was 8.1 months compared to 4.8 months with other cytotoxic chemotherapy regimens (Hazard ratio 0.61; 95% confidential interval 0.32-1.07).


MASTER KEY Project is one of the largest platform trials focused only on rare cancers. The project provided the chance for rare cancer patients to receive biomarker-based treatment, and it showed better prognosis. This is a continuous project aimed to accelerate treatment development for rare cancers, with clinical trials directed towards new drug approvals.

Clinical trial identification


Editorial acknowledgement

Legal entity responsible for the study

The authors.


Astellas Pharma, Bristol-Myers Squibb, Chugai Pharmaceutical, Daiichi Sankyo, Eisai, Kyorin Pharmaceutical, Novartis Parma, Ono Pharmaceutical, Otsuka Pharmaceutical, Pfizer, Taiho Pharmaceutical and Takeda Pharmaceutical.


All authors have declared no conflicts of interest.

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