{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,30]],"date-time":"2026-06-30T07:37:01Z","timestamp":1782805021365,"version":"3.54.5"},"reference-count":17,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2020,4,7]],"date-time":"2020-04-07T00:00:00Z","timestamp":1586217600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100007132","name":"Otsuka Pharmaceutical","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100007132","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004319","name":"Pfizer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004319","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["ascpt.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Clin Pharma and Therapeutics"],"published-print":{"date-parts":[[2020,9]]},"abstract":"<jats:p>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\u00a0=\u00a053) 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\u2010generation sequencing tests, with 48% (176\/364) harboring an \u201cactionable\u201d alteration. Seventy\u2010one (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\u2010directed or non\u2010biomarker\u2010directed 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.<\/jats:p>","DOI":"10.1002\/cpt.1817","type":"journal-article","created":{"date-parts":[[2020,2,29]],"date-time":"2020-02-29T13:01:01Z","timestamp":1582981261000},"page":"596-605","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":35,"title":["MASTER KEY Project: Powering Clinical Development for Rare Cancers Through a Platform Trial"],"prefix":"10.1111","volume":"108","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9639-7361","authenticated-orcid":false,"given":"Hitomi S.","family":"Okuma","sequence":"first","affiliation":[{"name":"Department of Breast and Medical Oncology Clinical Research Support Office National Cancer Center Hospital Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kan","family":"Yonemori","sequence":"additional","affiliation":[{"name":"Department of Breast and Medical Oncology Rare Cancer Center National Cancer Center Hospital Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shoko N.","family":"Narita","sequence":"additional","affiliation":[{"name":"Clinical Research Support Office National Cancer Center Hospital Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tamie","family":"Sukigara","sequence":"additional","affiliation":[{"name":"Clinical Research Support Office National Cancer Center Hospital Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Akihiro","family":"Hirakawa","sequence":"additional","affiliation":[{"name":"Department of Biostatistics and Bioinformatics Graduate School of Medicine The University of Tokyo Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Toshio","family":"Shimizu","sequence":"additional","affiliation":[{"name":"Department of Experimental Therapeutics National Cancer Center Hospital Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Taro","family":"Shibata","sequence":"additional","affiliation":[{"name":"Biostatistics Division, Center for Research Administration and Support National Cancer Center Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Akira","family":"Kawai","sequence":"additional","affiliation":[{"name":"Department of Musculoskeletal Oncology Rare Cancer Center National Cancer Center Hospital Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Noboru","family":"Yamamoto","sequence":"additional","affiliation":[{"name":"Department of Experimental Therapeutics Department of Thoracic Oncology National Cancer Center Hospital Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kenichi","family":"Nakamura","sequence":"additional","affiliation":[{"name":"Clinical Research Support Office National Cancer Center Hospital Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Toshiro","family":"Nishida","sequence":"additional","affiliation":[{"name":"Department of Surgery National Cancer Center Hospital Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasuhiro","family":"Fujiwara","sequence":"additional","affiliation":[{"name":"Department of Breast and Medical Oncology National Cancer Center Hospital Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,4,7]]},"reference":[{"key":"e_1_2_11_1_1","unstructured":"National Cancer Institute: Molecular Analysis for Therapy Choice (NCIMATCH<https:\/\/www.cancer.gov\/about\u2010cancer\/treatment\/clinical\u2010trials\/nci\u2010supported\/nci\u2010match>. Accessed July 31 2019."},{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMc1705868"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10637-011-9638-0"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/cpt.1136"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/cpt.1453"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/cas.13730"},{"key":"e_1_2_11_7_1","volume-title":"RARECARENet"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/cas.13969"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jmoldx.2016.10.002"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMsb1607705"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1038\/537S63a"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2016.11076"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.5694\/mja18.00227"},{"key":"e_1_2_11_14_1","unstructured":"Pharmaceuticals and Medical Devices Agency<https:\/\/www.pmda.go.jp\/english\/>. Accessed July 24 2019"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.1080\/19466315.2016.1173582"},{"key":"e_1_2_11_16_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2007.14.8114"},{"key":"e_1_2_11_17_1","volume-title":"Rare Cancers Japan"}],"container-title":["Clinical Pharmacology &amp; Therapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fcpt.1817","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/cpt.1817","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/cpt.1817","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/ascpt.onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/cpt.1817","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,11,7]],"date-time":"2023-11-07T13:37:38Z","timestamp":1699364258000},"score":1,"resource":{"primary":{"URL":"https:\/\/ascpt.onlinelibrary.wiley.com\/doi\/10.1002\/cpt.1817"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,4,7]]},"references-count":17,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2020,9]]}},"alternative-id":["10.1002\/cpt.1817"],"URL":"https:\/\/doi.org\/10.1002\/cpt.1817","archive":["Portico"],"relation":{},"ISSN":["0009-9236","1532-6535"],"issn-type":[{"value":"0009-9236","type":"print"},{"value":"1532-6535","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,4,7]]},"assertion":[{"value":"2019-10-28","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-01-22","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-04-07","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}