{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T13:14:06Z","timestamp":1793366046049,"version":"4.5.0"},"reference-count":25,"publisher":"SAGE Publications","issue":"6","license":[{"start":{"date-parts":[[2022,7,22]],"date-time":"2022-07-22T00:00:00Z","timestamp":1658448000000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100000265","name":"medical research council","doi-asserted-by":"publisher","award":["PR-OD-1017-20007"],"award-info":[{"award-number":["PR-OD-1017-20007"]}],"id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000060","name":"National Institute of Allergy and Infectious Diseases","doi-asserted-by":"publisher","award":["R01 AI 139761"],"award-info":[{"award-number":["R01 AI 139761"]}],"id":[{"id":"10.13039\/100000060","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000060","name":"National Institute of Allergy and Infectious Diseases","doi-asserted-by":"publisher","award":["R01 AI136947"],"award-info":[{"award-number":["R01 AI136947"]}],"id":[{"id":"10.13039\/100000060","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000060","name":"National Institute of Allergy and Infectious Diseases","doi-asserted-by":"publisher","award":["R37 AI 29168"],"award-info":[{"award-number":["R37 AI 29168"]}],"id":[{"id":"10.13039\/100000060","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Clinical Trials"],"published-print":{"date-parts":[[2022,12]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>The threat of a possible Marburg virus disease outbreak in Central and Western Africa is growing. While no Marburg virus vaccines are currently available for use, several candidates are in the pipeline. Building on knowledge and experiences in the designs of vaccine efficacy trials against other pathogens, including SARS-CoV-2, we develop designs of randomized Phase 3 vaccine efficacy trials for Marburg virus vaccines.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>A core protocol approach will be used, allowing multiple vaccine candidates to be tested against controls. The primary objective of the trial will be to evaluate the effect of each vaccine on the rate of virologically confirmed Marburg virus disease, although Marburg infection assessed via seroconversion could be the primary objective in some cases. The overall trial design will be a mixture of individually and cluster-randomized designs, with individual randomization done whenever possible. Clusters will consist of either contacts and contacts of contacts of index cases, that is, ring vaccination, or other transmission units.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>The primary efficacy endpoint will be analysed as a time-to-event outcome. A vaccine will be considered successful if its estimated efficacy is greater than 50% and has sufficient precision to rule out that true efficacy is less than 30%. This will require approximately 150 total endpoints, that is, cases of confirmed Marburg virus disease, per vaccine\/comparator combination. Interim analyses will be conducted after 50 and after 100 events. Statistical analysis of the trial will be blended across the different types of designs. Under the assumption of a 6-month attack rate of 1% of the participants in the placebo arm for both the individually and cluster-randomized populations, the most likely sample size is about 20,000 participants per arm.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>This event-driven design takes into the account the potentially sporadic spread of Marburg virus. The proposed trial design may be applicable for other pathogens against which effective vaccines are not yet available.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/17407745221110880","type":"journal-article","created":{"date-parts":[[2022,7,22]],"date-time":"2022-07-22T07:47:56Z","timestamp":1658476076000},"page":"647-654","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":22,"title":["A platform trial design for preventive vaccines against Marburg virus and other emerging infectious disease threats"],"prefix":"10.1177","volume":"19","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2780-9382","authenticated-orcid":false,"given":"Ira M","family":"Longini","sequence":"first","affiliation":[{"name":"Department of Biostatistics, University of Florida, Gainesville, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yang","family":"Yang","sequence":"additional","affiliation":[{"name":"Department of Biostatistics, University of Florida, Gainesville, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0420-5068","authenticated-orcid":false,"given":"Thomas R","family":"Fleming","sequence":"additional","affiliation":[{"name":"Department of Biostatistics, University of Washington, Seattle, WA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C\u00e9sar","family":"Mu\u00f1oz-Fontela","sequence":"additional","affiliation":[{"name":"Bernhard-Nocht-Institute for Tropical Medicine, Hamburg, Germany"},{"name":"German Center for Infection Research, DZIF, Partner site Hamburg, Hamburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rui","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Population Medicine, Harvard Pilgrim Health Care Institute and Harvard Medical School, Boston, MA, USA"},{"name":"Department of Biostatistics, Harvard University, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2364-5253","authenticated-orcid":false,"given":"Susan S","family":"Ellenberg","sequence":"additional","affiliation":[{"name":"Department of Biostatistics, Epidemiology, and Informatics, University of Pennsylvania, Philadelphia, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"George","family":"Qian","sequence":"additional","affiliation":[{"name":"London School of Hygiene & Tropical Medicine, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M Elizabeth","family":"Halloran","sequence":"additional","affiliation":[{"name":"Department of Biostatistics, University of Washington, Seattle, WA, USA"},{"name":"Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Center, Seattle, WA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martha","family":"Nason","sequence":"additional","affiliation":[{"name":"Biostatistics Research Branch, National Institute of Allergy and Infectious Diseases (NIAID\/NIH), Bethesda, MD, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2772-6242","authenticated-orcid":false,"given":"Victor De","family":"Gruttola","sequence":"additional","affiliation":[{"name":"Department of Biostatistics, Harvard University, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabue","family":"Mulangu","sequence":"additional","affiliation":[{"name":"Institut National de Recherche Biom\u00e9dicale, Kinshasa, Democratic Republic of the Congo"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yunda","family":"Huang","sequence":"additional","affiliation":[{"name":"London School of Hygiene & Tropical Medicine, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christl A","family":"Donnelly","sequence":"additional","affiliation":[{"name":"Department of Statistics, University of Oxford, Oxford, UK"},{"name":"Department of Infectious Disease Epidemiology, Imperial College London, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana-Maria","family":"Henao Restrepo","sequence":"additional","affiliation":[{"name":"World Health Organization, Geneva, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2022,7,22]]},"reference":[{"key":"bibr1-17407745221110880","volume":"374","author":"Mahase E","year":"2021","journal-title":"BMJ"},{"key":"bibr2-17407745221110880","unstructured":"World Health Organization. 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