{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T15:38:19Z","timestamp":1792165099728,"version":"4.4.0"},"reference-count":23,"publisher":"The Endocrine Society","issue":"6","license":[{"start":{"date-parts":[[2026,1,3]],"date-time":"2026-01-03T00:00:00Z","timestamp":1767398400000},"content-version":"vor","delay-in-days":1,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Soleno Therapeutics."}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,5,19]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Context<\/jats:title>\n                    <jats:p>The hallmark condition of Prader-Willi syndrome, a rare, genetic neurobehavioral\/metabolic disorder, is life-threatening hyperphagia.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>We assessed the efficacy and safety of diazoxide choline extended-release (DCCR) tablets for the treatment of hyperphagia in adults and children four years of age and older with Prader-Willi syndrome.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We conducted a 16-week, randomized withdrawal study in children and adults with Prader-Willi syndrome and hyperphagia. Participants who previously completed randomized (13-week DCCR or placebo) and open-label (2.5-4.5 years DCCR) studies were randomized 1:1 to receive once-daily DCCR or placebo. The primary endpoint was Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score change from baseline to 16 weeks. Secondary endpoints included Clinical Global Impression of Severity (CGI-S) and Improvement (CGI-I); exploratory endpoints included weight and body mass index (BMI) z-score.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Seventy-seven participants were randomized (DCCR:38; placebo:39). Statistically significant increases in HQ-CT from baseline to week 16 were observed with placebo vs DCCR [least square (LS) mean (standard error) change 7.6 (1.09)] with placebo and 2.6 (1.12) with DCCR; P = .0022. CGI scores favored DCCR but were not significantly changed. Consistent with the hyperphagia response, the placebo cohort gained more weight and increased their BMI z-score more than the DCCR cohort [LS mean weight difference (95% confidence interval) \u22121.6\u2005kg (\u22123.1, \u22120.1)]; LS mean z-score difference was \u22120.09 (\u22120.17, \u22120.01). Adverse events were similar with both treatments, with no serious adverse events in the DCCR arm.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Continued DCCR treatment was superior to placebo for hyperphagia. DCCR appears to offer meaningful therapeutic benefits for people with Prader-Willi syndrome.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1210\/clinem\/dgaf661","type":"journal-article","created":{"date-parts":[[2025,12,31]],"date-time":"2025-12-31T12:44:26Z","timestamp":1767185066000},"page":"e1554-e1561","source":"Crossref","is-referenced-by-count":9,"title":["Diazoxide Choline Extended-release Tablets in Prader-Willi Syndrome: A Randomized, Double-blind, Withdrawal Period Study"],"prefix":"10.1210","volume":"111","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4370-3438","authenticated-orcid":false,"given":"Jennifer L","family":"Miller","sequence":"first","affiliation":[{"name":"Department of Pediatric Endocrinology, University of Florida College of Medicine , Gainesville, FL 32608 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicola","family":"Bridges","sequence":"additional","affiliation":[{"name":"Department of Endocrinology, Chelsea and Westminster Hospital , London SW10 9NH ,","place":["UK"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric I","family":"Felner","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Emory University School of Medicine , Atlanta, GA 30322 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Parisa","family":"Salehi","sequence":"additional","affiliation":[{"name":"Endocrinology, Seattle Children's Hospital , Seattle, WA 98105 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jack A","family":"Yanovski","sequence":"additional","affiliation":[{"name":"US Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health , Bethesda, MD 20892 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David A","family":"Stevenson","sequence":"additional","affiliation":[{"name":"Department of Clinical Genetics, Stanford University , Palo Alto, CA 94305 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jorge","family":"Mejia-Corletto","sequence":"additional","affiliation":[{"name":"Department of Pediatric Endocrinology, NYU Langone Health , Mineola, NY 11501 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohamad G","family":"Shaikh","sequence":"additional","affiliation":[{"name":"Department of Endocrinology, Royal Hospital for Children , Glasgow G51 4TF ,","place":["UK"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M Jennifer","family":"Abuzzahab","sequence":"additional","affiliation":[{"name":"Department of Endocrinology, Children's Minnesota , St. Paul, MN 55102 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy","family":"Fleischman","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, Boston Children's Hospital , Boston, MA 02115 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1567-4449","authenticated-orcid":false,"given":"Virginia","family":"Kimonis","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, University of California, Irvine, and Children\u2019s Hospital of Orange County , Orange, CA 92868 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashley H","family":"Shoemaker","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Vanderbilt University Medical Center , Nashville, TN 37212 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anthony","family":"Holland","sequence":"additional","affiliation":[{"name":"Department of Psychiatry, Cambridgeshire & Peterborough NHS Foundation Trust, Fulbourn Hospital , Cambridge CB21 5EF ,","place":["UK"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lynne M","family":"Bird","sequence":"additional","affiliation":[{"name":"Department of Genetics, University of California, San Diego\/Rady Childrens Hospital , San Diego, CA 92123 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn S","family":"Obrynba","sequence":"additional","affiliation":[{"name":"Endocrinology, Nationwide Children's Hospital , Columbus, OH 43205 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa","family":"Lah","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Indiana University School of Medicine , Indianapolis, IN 46202 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth","family":"Littlejohn","sequence":"additional","affiliation":[{"name":"Department of Pediatric Endocrinology, University of Michigan Health\u2014Sparrow Clinical Research Institute , Lansing, MI 48912 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katerina","family":"Harwood","sequence":"additional","affiliation":[{"name":"Department of Pediatric Endocrinology, St. Joseph's University Medical Center , Paterson, NJ 07503 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heidi","family":"Shea","sequence":"additional","affiliation":[{"name":"Endocrinology, Research Institute of Dallas , Dallas, TX 75231 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Viskochil","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Utah , Salt Lake City, UT 84108 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patricia","family":"Hirano","sequence":"additional","affiliation":[{"name":"Soleno Therapeutics , Redwood City, CA 94065 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristen","family":"Yen","sequence":"additional","affiliation":[{"name":"Soleno Therapeutics , Redwood City, CA 94065 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shaila","family":"Ballal","sequence":"additional","affiliation":[{"name":"Soleno Therapeutics , Redwood City, CA 94065 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Huang","sequence":"additional","affiliation":[{"name":"Soleno Therapeutics , Redwood City, CA 94065 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neil M","family":"Cowen","sequence":"additional","affiliation":[{"name":"Soleno Therapeutics , Redwood City, CA 94065 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anish","family":"Bhatnagar","sequence":"additional","affiliation":[{"name":"Soleno Therapeutics , Redwood City, CA 94065 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4397-4126","authenticated-orcid":false,"given":"Evelien","family":"Gevers","sequence":"additional","affiliation":[{"name":"Endocrinology, Queen Mary University London , London E1 4NS ,","place":["UK"]},{"name":"Endocrinology, Barts Health NHS Trust-Royal London Children's Hospital , London E1 1FR ,","place":["UK"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"80","published-online":{"date-parts":[[2026,1,2]]},"reference":[{"issue":"2","key":"2026051819574363500_dgaf661-B1","doi-asserted-by":"crossref","first-page":"371","DOI":"10.1002\/ajmg.a.36845","article-title":"A population-based profile of 160 Australians with Prader-Willi syndrome: trends in diagnosis, birth prevalence and birth characteristics","volume":"167A","author":"Lionti","year":"2015","journal-title":"Am J Med Genet A"},{"issue":"7","key":"2026051819574363500_dgaf661-B2","doi-asserted-by":"crossref","first-page":"795","DOI":"10.1038\/s10038-018-0451-x","article-title":"An estimation of the prevalence of genomic disorders using chromosomal microarray data","volume":"63","author":"Gillentine","year":"2018","journal-title":"J Hum Genet"},{"issue":"5","key":"2026051819574363500_dgaf661-B3","doi-asserted-by":"crossref","first-page":"1040","DOI":"10.1002\/ajmg.a.33951","article-title":"Nutritional phases in Prader-Willi syndrome","volume":"155","author":"Miller","year":"2011","journal-title":"Am J Med Genet A"},{"issue":"1","key":"2026051819574363500_dgaf661-B4","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1542\/peds.2010-2820","article-title":"Clinical report-health supervision for children with Prader-Willi syndrome","volume":"127","author":"McCandless","year":"2011","journal-title":"Pediatrics"},{"issue":"1","key":"2026051819574363500_dgaf661-B5","doi-asserted-by":"crossref","first-page":"104379","DOI":"10.1016\/j.ejmg.2021.104379","article-title":"The consequences of hyperphagia in people with Prader-Willi syndrome: a systematic review of studies of morbidity and mortality","volume":"65","author":"Bellis","year":"2022","journal-title":"Eur J Med Genet"},{"issue":"6","key":"2026051819574363500_dgaf661-B6","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1038\/gim.2016.178","article-title":"Causes of death in prader-willi syndrome: Prader-Willi syndrome association (USA) 40-year mortality survey","volume":"19","author":"Butler","year":"2017","journal-title":"Genet Med"},{"issue":"3","key":"2026051819574363500_dgaf661-B7","doi-asserted-by":"crossref","first-page":"e0248739","DOI":"10.1371\/journal.pone.0248739","article-title":"Characteristics and relationship between hyperphagia, anxiety, behavioral challenges and caregiver burden in Prader-Willi syndrome","volume":"16","author":"Kayadjanian","year":"2021","journal-title":"PLoS One"},{"issue":"5","key":"2026051819574363500_dgaf661-B8","doi-asserted-by":"crossref","first-page":"417","DOI":"10.1111\/jir.13223","article-title":"Anxiety, depression and stress in parents and siblings of people who have Prader-Willi syndrome: morbidity prevalence and mitigating factors","volume":"69","author":"Micallef Pul\u00e8","year":"2025","journal-title":"J Intellect Disabil Res"},{"issue":"12","key":"2026051819574363500_dgaf661-B9","doi-asserted-by":"crossref","first-page":"4916","DOI":"10.1172\/JCI58035","article-title":"Activation of KATP channels suppresses glucose production in humans","volume":"121","author":"Kishore","year":"2011","journal-title":"J Clin Invest"},{"issue":"7","key":"2026051819574363500_dgaf661-B10","doi-asserted-by":"crossref","first-page":"1676","DOI":"10.1210\/clinem\/dgad014","article-title":"Diazoxide choline extended-release table in people with Prader-Willi syndrome: a double-blind, placebo-controlled trial","volume":"108","author":"Miller","year":"2023","journal-title":"J Clin Endocrinol Metab"},{"issue":"2","key":"2026051819574363500_dgaf661-B11","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1002\/oby.23928","article-title":"Diazoxide choline extended-release tablet in people with Prader-Willi syndrome: results from long-term open-label study","volume":"32","author":"Miller","year":"2024","journal-title":"Obesity (Silver Spring)"},{"issue":"1","key":"2026051819574363500_dgaf661-B12","doi-asserted-by":"crossref","first-page":"18614","DOI":"10.1038\/srep18614","article-title":"Snord116 is critical in the regulation of food intake and body weight","volume":"6","author":"Qi","year":"2016","journal-title":"Sci Rep"},{"issue":"1","key":"2026051819574363500_dgaf661-B13","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1172\/JCI88648","article-title":"Deficiency in prohormone convertase PC1 impairs prohormone processing in Prader-Willi syndrome","volume":"127","author":"Burnett","year":"2017","journal-title":"J Clin Invest"},{"issue":"4","key":"2026051819574363500_dgaf661-B14","doi-asserted-by":"crossref","first-page":"450","DOI":"10.3390\/genes11040450","article-title":"The potential role of activating the ATP-sensitive potassium channel in the treatment of hyperphagic obesity","volume":"11","author":"Cowen","year":"2020","journal-title":"Genes (Basel)"},{"issue":"4","key":"2026051819574363500_dgaf661-B15","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1016\/j.ymgme.2018.02.018","article-title":"Chronic diazoxide treatment decreases fat mass and improves endurance capacity in an obese mouse model of Prader-Willi syndrome","volume":"123","author":"Bischof","year":"2018","journal-title":"Mol Genet Metab"},{"issue":"1","key":"2026051819574363500_dgaf661-B16","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1186\/s11689-024-09536-x","article-title":"Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study","volume":"16","author":"Strong","year":"2024","journal-title":"J Neurodev Disord"},{"issue":"3","key":"2026051819574363500_dgaf661-B17","doi-asserted-by":"crossref","first-page":"PA25","DOI":"10.1016\/j.jval.2015.03.154","article-title":"Development of the hyperphagia questionnaire for use in Prader-Willi syndrome clinical trials","volume":"18","author":"Fehnel","year":"2015","journal-title":"Value Health"},{"issue":"1","key":"2026051819574363500_dgaf661-B18","doi-asserted-by":"crossref","first-page":"83","DOI":"10.1186\/s13023-024-03045-9","article-title":"The Prader-Willi syndrome profile: validation of a new measure of behavioral and emotional problems in Prader-Willi syndrome","volume":"19","author":"Dykens","year":"2024","journal-title":"Orphanet J Rare Dis"},{"key":"2026051819574363500_dgaf661-B19","unstructured":"Miller JL, Bridges N, Felner EI, et al Supplementary Materials for \u201cDiazoxide Choline Extended-release Tablets in Prader-Willi Syndrome: A Randomized, Double-blind, Withdrawal Period Study\u201d. Figshare. Deposited 9 January 2026. 10.6084\/m9.figshare.31038112.v2"},{"issue":"1","key":"2026051819574363500_dgaf661-B20","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1210\/clinem\/dgac549","article-title":"Liraglutide for weight management in children and adolescents with Prader-Willi syndrome and obesity","volume":"108","author":"Diene","year":"2023","journal-title":"J Clin Endocrinol Metab"},{"issue":"7","key":"2026051819574363500_dgaf661-B21","doi-asserted-by":"crossref","first-page":"1696","DOI":"10.1210\/clinem\/dgad015","article-title":"Intranasal carbetocin reduces hyperphagia, anxiousness and distress in Prader-Willi syndrome: CARE-PWS phase 3 trial","volume":"108","author":"Roof","year":"2023","journal-title":"J Clin Endocrinol Metab"},{"issue":"6","key":"2026051819574363500_dgaf661-B22","doi-asserted-by":"crossref","first-page":"e2373","DOI":"10.1210\/clinem\/dgac105","article-title":"The efficacy, safety, and pharmacology of a ghrelin o-acyltransferase inhibitor for the treatment of Prader-Willi syndrome","volume":"107","author":"Miller","year":"2022","journal-title":"J Clin Endocrinol Metab"},{"issue":"3","key":"2026051819574363500_dgaf661-B23","doi-asserted-by":"crossref","first-page":"2150","DOI":"10.3390\/ijms24032150","article-title":"Clinical trials in Prader-Willi syndrome: a review","volume":"24","author":"Mahmoud","year":"2023","journal-title":"Int J Mol Sci"}],"container-title":["The Journal of Clinical Endocrinology &amp; Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/academic.oup.com\/jcem\/advance-article-pdf\/doi\/10.1210\/clinem\/dgaf661\/66225244\/dgaf661.pdf","content-type":"application\/pdf","content-version":"am","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/jcem\/article-pdf\/111\/6\/e1554\/66225244\/dgaf661.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/jcem\/article-pdf\/111\/6\/e1554\/66225244\/dgaf661.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,18]],"date-time":"2026-05-18T23:57:50Z","timestamp":1779148670000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/jcem\/article\/111\/6\/e1554\/8413118"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,1,2]]},"references-count":23,"journal-issue":{"issue":"6","published-online":{"date-parts":[[2026,1,2]]},"published-print":{"date-parts":[[2026,5,19]]}},"URL":"https:\/\/doi.org\/10.1210\/clinem\/dgaf661","relation":{},"ISSN":["0021-972X","1945-7197"],"issn-type":[{"value":"0021-972X","type":"print"},{"value":"1945-7197","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2026,6]]},"published":{"date-parts":[[2026,1,2]]}}}