{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T10:48:46Z","timestamp":1790765326054,"version":"4.1.0"},"reference-count":32,"publisher":"SAGE Publications","issue":"3","license":[{"start":{"date-parts":[[2025,3,4]],"date-time":"2025-03-04T00:00:00Z","timestamp":1741046400000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2025,3,4]],"date-time":"2025-03-04T00:00:00Z","timestamp":1741046400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2025,5]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Facioscapulohumeral muscular dystrophy (FSHD) is a hereditary muscle disease without an available cure. The first trials with potentially disease-modifying therapies have started, including a phase ll open-label study and a phase lll double-blind randomized placebo-controlled trial assessing the safety and efficacy of losmapimod. Having a more in-depth understanding of the patient's experience of these trials will further enhance the design and recruitment of future trials.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>To explore the motivation, expectations, concerns, and experiences of FSHD patients in the first clinical trials in the Netherlands resulting in recommendations for future trials.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>Semi-structured interviews with participants of phase II and III losmapimod trials were conducted. The interview guide was based on previous conducted literature reviews and consultation of a patient representative. Participants were selected through convenience sampling. Four main themes were discussed: motivation for participation, expectations regarding study drug and trial visits, trial participation experience, and recommendations for future trials. The interviews were transcribed, anonymized, and analyzed using Atlas.ti version 23.1.1 using a deductive approach.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Thirteen participants were interviewed; six phase II participants and seven phase III participants. The primary motivations to participate concerned altruistic motives, contribute to science or improve their own health status. The participants had realistic expectations of the effect of the study drug before trial participation. Overall, participants were positive about their trial participation. Specifically, the personal and transparent communication within a trusting and dedicated trial team was appreciated. The phase III participants reported a higher than expected psychological burden on participating in a placebo-controlled trial. Recommendations consisted of more frequent updates on the overall progress and results of the trials.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>This study presents the participants\u2019 perspective on FSHD trials, providing important key findings for future clinical trial design, study site practices and patient education.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/22143602241313117","type":"journal-article","created":{"date-parts":[[2025,3,4]],"date-time":"2025-03-04T14:11:38Z","timestamp":1741097498000},"page":"382-393","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":3,"title":["The participants\u2019 perspective on facioscapulohumeral muscular dystrophy trials in The Netherlands \u2013 A qualitative study"],"prefix":"10.1177","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0009-0003-7180-484X","authenticated-orcid":false,"given":"Lizan","family":"Stinissen","sequence":"first","affiliation":[{"name":"Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, P.O. Box 9101, 6500 HB, Nijmegen, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joost","family":"Kools","sequence":"additional","affiliation":[{"name":"Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, P.O. Box 9101, 6500 HB, Nijmegen, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sietse","family":"Bouma","sequence":"additional","affiliation":[{"name":"Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, P.O. Box 9101, 6500 HB, Nijmegen, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma","family":"Lenssen","sequence":"additional","affiliation":[{"name":"Clinical Research Unit, Radboudumc, Nijmegen, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eline","family":"Sanders","sequence":"additional","affiliation":[{"name":"Clinical Research Unit, Radboudumc, Nijmegen, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anke","family":"Lanser","sequence":"additional","affiliation":[{"name":"Dutch FSHD patient advocacy group, Wassenaar, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ria","family":"de Haas","sequence":"additional","affiliation":[{"name":"FSHD Europe, Den Haag, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Baziel GM","family":"van Engelen","sequence":"additional","affiliation":[{"name":"Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, P.O. Box 9101, 6500 HB, Nijmegen, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wija","family":"Oortwijn","sequence":"additional","affiliation":[{"name":"Research Institute for Medical Innovation, Science department IQ Health, Radboud University Medical Center, P.O. Box 9101, 6500 HB, Nijmegen, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5837-7295","authenticated-orcid":false,"given":"Nicol C","family":"Voermans","sequence":"additional","affiliation":[{"name":"Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, P.O. Box 9101, 6500 HB, Nijmegen, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2025,3,4]]},"reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000797"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1017\/cjn.2015.311"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1136\/practneurol-2015-001353"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.20522"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000001783"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.molmed.2011.01.001"},{"key":"e_1_3_5_8_2","unstructured":"FSHD Society. Drug Development Pipeline [Internet]. [cited 2024 July 16] Available from: https:\/\/www.fshdsociety.org\/therapeutic-accelerator\/drug-development-pipeline\/."},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2024.123096"},{"key":"e_1_3_5_10_2","unstructured":"Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH). ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000 Feb 29 - . Identifier NCT05397470 Facioscapulohumeral Dystrophy (FSHD) [cited 2023 Jul 19]; Available from: https:\/\/clinicaltrials.gov\/study\/NCT05397470?cond=FSHD&rank=2."},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1186\/s12883-019-1452-x"},{"key":"e_1_3_5_12_2","doi-asserted-by":"publisher","DOI":"10.1111\/cge.13446"},{"key":"e_1_3_5_13_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.clinthera.2024.03.008"},{"key":"e_1_3_5_14_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2023.04.001"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2023.12.014"},{"key":"e_1_3_5_16_2","doi-asserted-by":"publisher","DOI":"10.1093\/intqhc\/mzm042"},{"key":"e_1_3_5_17_2","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.14884"},{"key":"e_1_3_5_18_2","doi-asserted-by":"publisher","DOI":"10.1124\/jpet.119.259663"},{"key":"e_1_3_5_19_2","unstructured":"Fulcrum Therapeutics Announces Topline Results from Phase 3 REACH Clinical Trial of Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD). Fulcrum Therapeutics. Accessed on: 12 Sep 2024. Available from:https:\/\/ir.fulcrumtx.com\/news-releases\/news-release-details\/fulcrum-therapeutics-announces-topline-results-phase-3-reach"},{"key":"e_1_3_5_20_2","doi-asserted-by":"publisher","DOI":"10.1007\/s43441-019-00033-1"},{"key":"e_1_3_5_21_2","doi-asserted-by":"publisher","DOI":"10.3768\/rtipress.2018.op.0054.1808"},{"key":"e_1_3_5_22_2","doi-asserted-by":"publisher","DOI":"10.1188\/21.CJON.647-654"},{"key":"e_1_3_5_23_2","doi-asserted-by":"publisher","DOI":"10.1191\/1478088706qp063oa"},{"key":"e_1_3_5_24_2","first-page":"149","article-title":"A critical review of qualitative interviews","volume":"3","author":"Alsaawi A","year":"2014","unstructured":"Alsaawi A. A critical review of qualitative interviews. 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Identifier NCT04060199 [cited 2024 27 jun] Available from: https:\/\/clinicaltrials.gov\/study\/NCT04060199?cond=NCT04060199&rank=1"},{"key":"e_1_3_5_28_2","doi-asserted-by":"publisher","DOI":"10.1002\/mds.29252"},{"key":"e_1_3_5_29_2","unstructured":"Society F. FSHD Society. FSHD Navigator [Internet]. Massachusettes (US)[cited 2024 July 16]. Available from: https:\/\/www.fshdsociety.org\/for-patients-families\/fshd-navigator\/."},{"key":"e_1_3_5_30_2","unstructured":"Avidity Biosciences. Fortitude study [Internet]. [cited 2024 July 16] Available from: https:\/\/fortitude-study.com\/."},{"key":"e_1_3_5_31_2","doi-asserted-by":"publisher","DOI":"10.1186\/s40900-020-00188-4"},{"key":"e_1_3_5_32_2","doi-asserted-by":"publisher","DOI":"10.1080\/14737167.2019.1612242"},{"key":"e_1_3_5_33_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2023.157"}],"container-title":["Journal of Neuromuscular Diseases"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/22143602241313117","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/22143602241313117","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/22143602241313117","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,1,6]],"date-time":"2026-01-06T06:39:41Z","timestamp":1767681581000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/22143602241313117"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,3,4]]},"references-count":32,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2025,5]]}},"alternative-id":["10.1177\/22143602241313117"],"URL":"https:\/\/doi.org\/10.1177\/22143602241313117","relation":{},"ISSN":["2214-3599","2214-3602"],"issn-type":[{"value":"2214-3599","type":"print"},{"value":"2214-3602","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,3,4]]}}}