{"doi":"10.1210/clinem/dgad014","title":"Diazoxide Choline Extended-Release Tablet in People With Prader-Willi Syndrome: A Double-Blind, Placebo-Controlled Trial","abstract":"CONTEXT: Prader-Willi syndrome (PWS) is a rare neurobehavioral-metabolic disease caused by the lack of paternally expressed genes in the chromosome 15q11-q13 region, characterized by hypotonia, neurocognitive problems, behavioral difficulties, endocrinopathies, and hyperphagia resulting in severe obesity if not controlled. OBJECTIVE: The primary end point was change from baseline in hyperphagia using the Hyperphagia Questionnaire for Clinical Trials (HQ-CT). Other end points included Global Impression Scores, and changes in body composition, behaviors, and hormones. METHODS: In DESTINY PWS, a 13-week, randomized, double-blind, placebo-controlled, phase 3 trial, 127 participants with PWS aged 4 years and older with hyperphagia were randomly assigned 2:1 to diazoxide choline extended-release tablet (DCCR) or placebo. RESULTS: DCCR did not significantly improve hyperphagia (HQ-CT least-square mean (LSmean) [SE] -5.94 [0.879] vs -4.27 [1.145]; P = .198), but did so in participants with severe hyperphagia (LSmean [SE] -9.67 [1.429] vs -4.26 [1.896]; P = .012). Two of 3 secondary end points were improved (Clinical Global Impression of Improvement [CGI-I]; P = .029; fat mass; P = .023). In an analysis of results generated pre-COVID, the primary (HQ-CT; P = .037) and secondary end points were all improved (CGI-I; P = .015; Caregiver Global Impression of Change; P = .031; fat mass; P = .003). In general, DCCR was well tolerated with 83.3% in the DCCR group experiencing a treatment-emergent adverse event and 73.8% in the placebo group (not significant). CONCLUSION: DCCR did not significantly improve hyperphagia in the primary analysis but did in participants with severe baseline hyperphagia and in the pre-COVID analysis. DCCR treatment was associated with significant improvements in body composition and clinician-reported outcomes.","journal":"The Journal of Clinical Endocrinology & Metabolism","year":2023,"id":320173,"datarank":1.3171422114760485,"base_score":3.9318256327243257,"endowment":3.9318256327243257,"self_citation_contribution":0.5897738449086489,"citation_network_contribution":0.7273683665673996,"self_endowment_contribution":0.5897738449086489,"citer_contribution":0.7273683665673996,"corpus_percentile":null,"corpus_rank":null,"citation_count":50,"citer_count":34,"citers_with_citation_signal":22,"citers_with_endowment":22,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9587,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT03440814"]},"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2023-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":1030630,"name":"Evelien Gevers","orcid":"0000-0002-4397-4126","position":1,"is_corresponding":false},{"id":1030631,"name":"Nicola Bridges","orcid":"0000-0002-2915-4877","position":2,"is_corresponding":false},{"id":402285,"name":"Jack A. Yanovski","orcid":"0000-0001-8542-1637","position":3,"is_corresponding":false},{"id":1030632,"name":"Parisa Salehi","orcid":"0000-0003-1586-6336","position":4,"is_corresponding":false},{"id":1030633,"name":"Kathryn Obrynba","orcid":"0000-0003-1443-577X","position":5,"is_corresponding":false},{"id":231482,"name":"Eric I. Felner","orcid":"0000-0002-6567-0123","position":6,"is_corresponding":false},{"id":249069,"name":"Lynne M. Bird","orcid":"0000-0003-4833-3747","position":7,"is_corresponding":false},{"id":701643,"name":"Ashley H. Shoemaker","orcid":"0000-0003-1628-3677","position":8,"is_corresponding":false},{"id":1031395,"name":"Moris Angulo","orcid":null,"position":9,"is_corresponding":false},{"id":326229,"name":"Merlin G. Butler","orcid":"0000-0002-2911-0524","position":10,"is_corresponding":false},{"id":633176,"name":"David A. Stevenson","orcid":"0000-0001-8601-0020","position":11,"is_corresponding":false},{"id":1031396,"name":"Jennifer Abuzzahab","orcid":null,"position":12,"is_corresponding":false},{"id":962195,"name":"Timothy Barrett","orcid":"0000-0002-6873-0750","position":13,"is_corresponding":false},{"id":1030634,"name":"Melissa Lah","orcid":"0000-0002-2406-8961","position":14,"is_corresponding":false},{"id":1031397,"name":"Elizabeth Littlejohn","orcid":null,"position":15,"is_corresponding":false},{"id":229947,"name":"Verghese Mathew","orcid":null,"position":16,"is_corresponding":false},{"id":1030635,"name":"Neil Cowen","orcid":"0000-0003-4189-1009","position":17,"is_corresponding":false},{"id":1030636,"name":"Anish Bhatnagar","orcid":"0000-0001-7078-6959","position":18,"is_corresponding":false},{"id":1031398,"name":"the DESTINY PWS Investigators","orcid":null,"position":19,"is_corresponding":false},{"id":1030637,"name":"Urmi Das","orcid":"0000-0003-3624-8780","position":20,"is_corresponding":false},{"id":1030638,"name":"Amy Fleischman","orcid":"0000-0002-5497-4367","position":21,"is_corresponding":false},{"id":876161,"name":"Anthony P. 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