{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T06:22:27Z","timestamp":1782973347460,"version":"3.54.5"},"reference-count":33,"publisher":"Wiley","issue":"19","license":[{"start":{"date-parts":[[2021,5,7]],"date-time":"2021-05-07T00:00:00Z","timestamp":1620345600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Statistics in Medicine"],"published-print":{"date-parts":[[2021,8,30]]},"abstract":"<jats:p>A Bayesian adaptive design is proposed for a clinical trial in Duchenne muscular dystrophy. The trial was designed to demonstrate treatment efficacy on an ambulatory\u2010based clinical endpoint and to identify early success on a biomarker (dystrophin protein levels) that can serve as a basis for accelerated approval in the United States. The trial incorporates placebo augmentation using placebo data from past clinical trials. A thorough simulation study was conducted to understand the operating characteristics of the trial. This trial design was selected for the US FDA Complex Innovative Trial Design Pilot Meeting Program and the experience in that program is summarized.<\/jats:p>","DOI":"10.1002\/sim.9021","type":"journal-article","created":{"date-parts":[[2021,5,8]],"date-time":"2021-05-08T21:24:33Z","timestamp":1620509073000},"page":"4167-4184","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Bayesian adaptive design for clinical trials in Duchenne muscular dystrophy"],"prefix":"10.1002","volume":"40","author":[{"given":"Stephen L.","family":"Lake","sequence":"first","affiliation":[{"name":"Wave Life Sciences Ltd  Cambridge Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melanie A.","family":"Quintana","sequence":"additional","affiliation":[{"name":"Berry Consultants LLC  Austin Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristine","family":"Broglio","sequence":"additional","affiliation":[{"name":"Berry Consultants LLC  Austin Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Panagoulias","sequence":"additional","affiliation":[{"name":"Wave Life Sciences Ltd  Cambridge Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Scott M.","family":"Berry","sequence":"additional","affiliation":[{"name":"Berry Consultants LLC  Austin Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael A.","family":"Panzara","sequence":"additional","affiliation":[{"name":"Wave Life Sciences Ltd  Cambridge Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2021,5,7]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(87)90579-4"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(88)90383-2"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/0960-8966(91)90039-U"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2014.03.008"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23528"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073814558120"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70272-8"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2007.03.002"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/pri.520"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0164684"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)31611-2"},{"key":"e_1_2_10_14_1","unstructured":"European Medicines Agency.Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy. December 17 2015 EMA\/CHMP\/236981\/2011 Corr. 11 Committee for Medicinal Products for Human Use (CHMP)."},{"key":"e_1_2_10_15_1","unstructured":"Complex innovative designs pilot meeting program (Notice).83 Federal Register 44274. August 30 2018."},{"key":"e_1_2_10_16_1","unstructured":"21st Century Cures Act. H.R. 34 114th Congress.2016.https:\/\/www.gpo.gov\/fdsys\/pkg\/BILLS-114hr34enr\/pdf\/BILLS-114hr34enr.pdf."},{"key":"e_1_2_10_17_1","unstructured":"Food and Drug Administration Center for Drug Evaluation and Research.Duchenne muscular dystrophy and related dystrophinopathies: developing drugs for treatment guidance for industry. 2018 FDA\u20102015\u2010D\u20101884."},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1080\/10543406.2014.888569"},{"key":"e_1_2_10_19_1","unstructured":"EXONDYS 51\u2122 [package insert].https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2016\/206488lbl.pdf."},{"key":"e_1_2_10_20_1","unstructured":"VILTEPSO\u2122 [package insert].https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2020\/212154s000lbl.pdf."},{"key":"e_1_2_10_21_1","unstructured":"VYONDYS 53\u2122 [package insert].https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2019\/211970s000lbl.pdf."},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.7811"},{"key":"e_1_2_10_23_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.8050"},{"key":"e_1_2_10_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.2044-8317.2011.02037.x"},{"key":"e_1_2_10_25_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1524-4733.2008.00347.x"},{"key":"e_1_2_10_26_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.4780142408"},{"key":"e_1_2_10_27_1","doi-asserted-by":"publisher","DOI":"10.1177\/096228020101000404"},{"key":"e_1_2_10_28_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000004570"},{"key":"e_1_2_10_29_1","unstructured":"CharnasL MarraffinoS.Domagrozumab (anti\u2010myostatin) as a potential treatment for Duchenne muscular dystrophy. Parent Project Muscular Dystrophy webinar. May 22 2019; Scottsdale AZ.https:\/\/www.parentprojectmd.org\/watch\u2010domagrozumab\u2010as\u2010a\u2010potential\u2010treatment\u2010for\u2010duchenne\u2010webinar\u2010recording"},{"key":"e_1_2_10_30_1","doi-asserted-by":"publisher","DOI":"10.1016\/0021-9681(76)90044-8"},{"key":"e_1_2_10_31_1","unstructured":"Food and Drug Administration Center for Drug Evaluation and Research.Center for Biologics Evaluation and Research. Rare diseases: common issues in drug development guidance for industry. 2019. FDA\u20102015\u2010D\u20102818."},{"key":"e_1_2_10_32_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000010170"},{"key":"e_1_2_10_33_1","doi-asserted-by":"publisher","DOI":"10.1201\/9781315180212-12"},{"key":"e_1_2_10_34_1","doi-asserted-by":"publisher","DOI":"10.1111\/biom.12841"}],"updated-by":[{"DOI":"10.1002\/sim.9378","type":"correction","label":"Correction","source":"publisher","updated":{"date-parts":[[2022,4,11]],"date-time":"2022-04-11T00:00:00Z","timestamp":1649635200000}}],"container-title":["Statistics in Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/sim.9021","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/sim.9021","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/sim.9021","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,29]],"date-time":"2023-08-29T04:01:23Z","timestamp":1693281683000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/sim.9021"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,5,7]]},"references-count":33,"journal-issue":{"issue":"19","published-print":{"date-parts":[[2021,8,30]]}},"alternative-id":["10.1002\/sim.9021"],"URL":"https:\/\/doi.org\/10.1002\/sim.9021","archive":["Portico"],"relation":{},"ISSN":["0277-6715","1097-0258"],"issn-type":[{"value":"0277-6715","type":"print"},{"value":"1097-0258","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,5,7]]},"assertion":[{"value":"2020-10-09","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-04-16","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-05-07","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}