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Early-phase clinical trials (EPCTs) may provide an actionable option as trial designs and precision medicine approaches evolve. A retrospective 5-year UK specialist-centre study evaluates referral, genomic profiling, EPCT participation, response, progression-free survival, overall survival, and prognostic correlations.",{"@graph":69,"@context":122},[70,84,105],{"@type":71,"itemListElement":72},"BreadcrumbList",[73,77,79,82],{"item":74,"name":75,"@type":76,"position":8},"https://docshare.wps.com","Home","ListItem",{"item":78,"name":9,"@type":76,"position":14},"https://docshare.wps.com/document/",{"item":80,"name":35,"@type":76,"position":81},"https://docshare.wps.com/document/healthcare/",3,{"item":83,"name":65,"@type":76,"position":19},"https://docshare.wps.com/document/a-single-centre-experience-of-patients-with-rare-cancers-referred-for-early-phase-clinical-trials/374276/",{"url":83,"name":65,"@type":85,"image":86,"author":91,"headline":65,"publisher":94,"fileFormat":97,"inLanguage":63,"description":67,"dateModified":98,"datePublished":99,"encodingFormat":97,"isAccessibleForFree":100,"interactionStatistic":101},"DigitalDocument",{"url":87,"@type":88,"width":89,"height":90},"https://docshare.wps.com/thumbnails/a-single-centre-experience-of-patients-with-rare-cancers-referred-for-early-phase-clinical-trials/374276.png","ImageObject",300,407,{"name":92,"@type":93},"Miles","Person",{"url":74,"name":95,"@type":96},"DocShare","Organization","application/pdf","2026-10-27","2026-09-23",true,{"@type":102,"interactionType":103,"userInteractionCount":52},"InteractionCounter",{"@type":104},"ViewAction",{"@type":106,"mainEntity":107},"FAQPage",[108,114,118],{"name":109,"@type":110,"acceptedAnswer":111},"How were patients with rare cancers selected for the study?","Question",{"text":112,"@type":113},"Patients with rare cancers referred to a large EPCT team at a UK specialist centre were included in a retrospective cohort spanning 2016–2020.","Answer",{"name":115,"@type":110,"acceptedAnswer":116},"What proportion of patients had molecular profiling and actionable gene alterations?",{"text":117,"@type":113},"Molecular profiling was offered to 45.5% of patients. Among those with identified gene aberrations, actionable gene alterations were reported in 60.2%.",{"name":119,"@type":110,"acceptedAnswer":120},"What outcomes were observed for patients who participated in early-phase clinical trials?",{"text":121,"@type":113},"Fifty-one patients participated in EPCTs, with 39.2% achieving stable disease and 11.8% achieving partial response. Median progression-free survival was three months, and median overall survival was 16 months for trial participants versus 7 months for non-trial participants.","https://schema.org",{"og:url":83,"og:type":124,"og:title":65,"og:site_name":95,"og:description":67},"article",{"robots":126,"canonical":83},"index,follow",{"doc_id":128,"site_id":62},374276,1791022942,{"code":4,"msg":5,"data":131},{"doc_id":128,"user_id":132,"nickname":92,"user_avatar":133,"doc_module":4,"category_id":34,"category_name":35,"doc_title":65,"doc_description":67,"doc_content":134,"file_id":135,"file_url":136,"file_type":137,"file_size":138,"view_count":52,"is_deleted":4,"is_public":8,"is_downloadable":8,"audit_status":8,"page_count":139,"language":140,"language_code":63,"site_id":62,"html_lang":63,"table_of_contents":141,"faqs":142,"seo_title":143,"seo_description":67,"update_tm":144,"read_time":41},13056703019404,"https://ap-avatar.wpscdn.com/davatar_29158cc5080c5b710cf443261637dec0","Angelakas et al. BMC Cancer (2025) 25:558 [https://doi.org/10.1186/s12885-025-13934-2](https://doi.org/10.1186/s12885-025-13934-2)  \nBMC Cancer  \n RESEARCH Open Access  \nA single centre experience of patients  \nwith rare cancers referred for early phase clinical trials  \nAngelos Angelakas2, Natalie Cook1,2, Donna M. Graham 1,2, Matthew Krebs1,2, Fiona Thistlethwaite1,2 and Louise Carter1,2*  \nAbstract  \nBackground Cancers affecting \u003C 6/100,000/year are classified as rare, but they account for up to 25% of all cancersand are associated with worse 5-year survival than common cancers. Early-phase clinical trials (EPCTs) may represent a viable treatment option for patients with rare cancers as they have evolved significantly with novel designsand the increasing use of precision medicine.  \nMethods A retrospective study of patients with rare cancers referred to a large EPCT team at a UK specialist centre over 5 years (2016–2020) was conducted. Patient demographics, medical and oncological history, genomic variants, EPCT participation, responses and survival outcomes were analysed.  \nResults In total, 240 patients with rare cancers were included. The mean age at diagnosis was 51.7 years (range 16–84), 54. 2% of the patients were female. The most frequent rare cancers originated from the digestive system (27 . 1%), female genital tract (20%) and head and neck (H + N) (18 . 3%) . Molecular profiling was offered to 45. 5% of the population, median number of gene alterations was 3 per patient (range 1–20) while actionable gene alterations were reported in 60.2%(n= 50) of those with identified gene aberrations. Fifty-one patients participated in EPCTs, with 39. 2% achieving SD and 11. 8% PR. Median PFS for trial participants was three months (95% CI 1.12 – 4. 88) while median OS in the trial patients was 16 months (95% CI 9.10 – 22. 90) compared to 7 months for non-trial participants (95% CI 5.50 – 8. 51) . Finally, poor Royal Marsden Hospital (RMH) prognostic score (2–3) was correlated with worse survival when controlling for age and sex (HR 1. 714, 95% CI 1.19 – 2.46, p = 0 . 004) .  \nConclusions Participation of patients with rare cancers in EPCTs may be associated with a survival benefit and lead to the development of new treatments for these patients. Moreover, expanded use of precision medicine is paramount as it can inform targeted treatment selection in this heterogenous group.  \nKeywords Rare cancers, Early phase trials, Real world data, Prognosis, Precision medicine, Molecular profiling, Targeted treatment  \n*Correspondence:  \nLouise Carter [louise.carter@manchester.ac.uk](louise.carter@manchester.ac.uk)  \n1 Division of Cancer Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK  \n2 Department of Medical Oncology, The Christie NHS Foundation Trust, Manchester, UK  \nBackground  \nRare cancers are a group of heterogeneous primary tumours which, although individually rare, collectively represent a significant proportion of all cancer diagnoses. In Europe, the United States (US) and the United Kingdom, rare cancers are estimated to account for 20%−24% of all cancers [1–4]. Developing an accepted definition  \n© The Author(s) 2025. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a c","cbCaiqT50wT7bj38","https://ap.wps.com/l/cbCaiqT50wT7bj38","pdf",1450301,12,"English","# Abstract\n## Background\n## Methods\n## Results\n## Conclusions\n# Keywords","[{\"question\":\"How were patients with rare cancers selected for the study?\",\"answer\":\"Patients with rare cancers referred to a large EPCT team at a UK specialist centre were included in a retrospective cohort spanning 2016–2020.\"},{\"question\":\"What proportion of patients had molecular profiling and actionable gene alterations?\",\"answer\":\"Molecular profiling was offered to 45.5% of patients. Among those with identified gene aberrations, actionable gene alterations were reported in 60.2%.\"},{\"question\":\"What outcomes were observed for patients who participated in early-phase clinical trials?\",\"answer\":\"Fifty-one patients participated in EPCTs, with 39.2% achieving stable disease and 11.8% achieving partial response. Median progression-free survival was three months, and median overall survival was 16 months for trial participants versus 7 months for non-trial participants.\"}]","A single centre experience of patients with rare cancers referred for early phase clinical trials | PDF",1790201397]