2f:I[703058,["/_next/static/immutable/chunks/3bhq_5eh1d760.js","/_next/static/immutable/chunks/1855-ogyi8eh4.js","/_next/static/immutable/chunks/2pfsjpn6k6zlg.js","/_next/static/immutable/chunks/26c7v3_3uwnwu.js","/_next/static/immutable/chunks/25a3ozcrvi1ff.js","/_next/static/immutable/chunks/2r4wx50nvjh_4.js","/_next/static/immutable/chunks/23fsw06hsi5b1.js","/_next/static/immutable/chunks/06_yt270h41rg.js","/_next/static/immutable/chunks/3ezsiz-7shkna.js","/_next/static/immutable/chunks/20x2y96ldu5wx.js","/_next/static/immutable/chunks/2lxntcm3pd1_h.js","/_next/static/immutable/chunks/00dgco8irq3jw.js","/_next/static/immutable/chunks/00nwe-kq0rboc.js","/_next/static/immutable/chunks/2051ffx3xzio9.js"],"PaperListPageClient"] 30:T71a,The hallmark condition of Prader-Willi syndrome, a rare, genetic neurobehavioral/metabolic disorder is life-threatening hyperphagia. We assessed the efficacy and safety of recently FDA-approved 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. 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 one:one 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. Seventy-seven participants were randomized (DCCR:38; placebo:39). Statistically significant increases in HQ-CT from baseline to week 16 were observed with placebo versus DCCR (least square [LS] mean [standard error] change 7.6 [1.09] with placebo and 2.6 [1.12] with DCCR; P=0.0022). 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) -1.6 kg (-3.1, -0.1); LS mean z-score difference -0.09 (-0.17, -0.01). Adverse events were similar with both treatments, with no serious adverse events in the DCCR arm. Continued DCCR treatment was superior to placebo for hyperphagia. DCCR appears to offer meaningful therapeutic benefits for people with Prader-Willi syndrome.31:Ta21,Abstract Context The hallmark condition of Prader-Willi syndrome, a rare, genetic neurobehavioral/metabolic disorder, is life-threatening hyperphagia. Objective 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. Methods 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. Results 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) −1.6 kg (−3.1, −0.1)]; LS mean z-score difference was −0.09 (−0.17, −0.01). Adverse events were similar with both treatments, with no serious adverse events in the DCCR arm. Conclusion Continued DCCR treatment was superior to placebo for hyperphagia. DCCR appears to offer meaningful therapeutic benefits for people with Prader-Willi syndrome. 25:[[["$","script","0",{"type":"application/ld+json","dangerouslySetInnerHTML":{"__html":"{\"@context\":\"https://schema.org\",\"@type\":\"CollectionPage\",\"name\":\"Prader-Willi Syndrome: condition Research & Clinical Takeaways\",\"description\":\"Explore peer-reviewed papers on Prader-Willi Syndrome (condition). Clinician-written summaries and actionable practice insights. 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What do the General Movements Assessment and Motor Optimality Score tell us about infants with developmental central hypotonia? A scoping review","journal_title":"Early Human Development","journal_issn":"0378-3782","published_at":"2026-05-14T00:00:00.000Z","url":"https://doi.org/10.1016/j.earlhumdev.2026.106583","source_score":4,"is_retracted":false,"is_open_access":true,"raw_metadata":{"pubmed":true,"crossref":true,"unpaywall":true},"created_at":"2026-05-21T13:20:57.658Z","updated_at":"2026-05-21T13:20:57.658Z","tags":[{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"age_group","value":"infant","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"category","value":"Obstetrics & Gynecology","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"condition","value":"Cornelia de Lange Syndrome","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"condition","value":"Cri du Chat Syndrome","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"condition","value":"Developmental Central Hypotonia","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"condition","value":"Down Syndrome","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"condition","value":"Hypotonic Cerebral Palsy","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"condition","value":"Prader-Willi Syndrome","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"condition","value":"Smith-Magenis Syndrome","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"condition","value":"West Syndrome","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"impact_flag","value":"limited_evidence","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"level_of_evidence","value":"5","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"modality","value":"Neurodevelopmental Assessment","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"sample_size","value":"n=14","value_numeric":14},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"study_design","value":"Scoping Review","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"warning","value":"Sample size tag reflects the number of included studies (n=14), not a participant-level N, as total participant count was not reported in the abstract.","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"warning","value":"Study design classified as 'Scoping Review' rather than 'Systematic Review' as stated by authors; no meta-analysis was performed.","value_numeric":null},{"paper_doi":"10.1016/j.earlhumdev.2026.106583","tag_type":"warning","value":"The 12 diagnoses/etiologies varied widely; pooled conclusions about 'developmental central hypotonia' as a single entity should be interpreted cautiously given clinical heterogeneity.","value_numeric":null}],"summaries":{"what_studied":"A scoping review mapping how Prechtl's General Movements Assessment (GMA) and the Motor Optimality Score-Revised (MOS-R) have been applied in infants with developmental central hypotonia (≤5 months corrected age), covering 14 studies across 12 diagnoses including Down syndrome, Prader-Willi syndrome, hypotonic cerebral palsy, and others.","key_result":"Across all conditions, spontaneous motor behavior showed reduced variability and complexity, a below-age-expected repertoire, atypical posture, and predominantly slow or monotonous movement; atypical fidgety patterns were frequent, though fidgety movements could still be present in Down syndrome and Prader-Willi syndrome infants.","limitations":"Most included studies were small and descriptive; evidence was heterogeneous across 12 different diagnoses; MOS-R subdomain reporting was not standardized across studies, limiting cross-study comparisons.","practice_takeaway":"GMA and MOS-R are feasible and reliable tools to phenotype early motor patterns in infants with developmental central hypotonia and can strengthen detection and surveillance pathways. Clinicians should consider integrating these tools into early monitoring, while awaiting prospective longitudinal data on their association with functional outcomes and responsiveness to early intervention."}},{"doi":"10.1210/clinem/dgaf661","pmid":"41482637","title":"Diazoxide Choline Extended-release Tablets in Prader-Willi Syndrome: A Randomized, Double-blind, Withdrawal Period Study","journal_title":"The Journal of Clinical Endocrinology & Metabolism","journal_issn":"0021-972X","published_at":"2026-01-03T00:00:00.000Z","url":"https://doi.org/10.1210/clinem/dgaf661","source_score":64,"is_retracted":false,"is_open_access":true,"raw_metadata":{"ooir":{"day":"2026-01-06","doi":"10.1210/clinem/dgaf661","days":"14","issn":"0021-972X","rank":"9","field":"Clinical Medicine","score":"64","category":"Endocrinology & Metabolism","categories":["Endocrinology & Metabolism"],"_categories":["Endocrinology & Metabolism"]},"pubmed":{"doi":"10.1210/clinem/dgaf661","title":"Diazoxide Choline Extended-Release Tablets in Prader-Willi Syndrome: A Randomized, Double-Blind, Withdrawal Period Study.","journal":"The Journal of clinical endocrinology and metabolism","methods":"","results":"Seventy-seven participants were randomized (DCCR:38; placebo:39). Statistically significant increases in HQ-CT from baseline to week 16 were observed with placebo versus DCCR (least square [LS] mean [standard error] change 7.6 [1.09] with placebo and 2.6 [1.12] with DCCR; P=0.0022). 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) -1.6 kg (-3.1, -0.1); LS mean z-score difference -0.09 (-0.17, -0.01). Adverse events were similar with both treatments, with no serious adverse events in the DCCR arm.","abstract":"$30","keywords":["Prader-Willi syndrome","diazoxide choline extended-release tablets","hyperphagia","randomized withdrawal period study"],"pubmed_id":"41482637","conclusions":"","publication_date":"2026-01-03T00:00:00"},"crossref":{"DOI":"10.1210/clinem/dgaf661","URL":"https://doi.org/10.1210/clinem/dgaf661","ISSN":["0021-972X","1945-7197"],"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/advance-article-pdf/doi/10.1210/clinem/dgaf661/66225244/dgaf661.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"page":null,"type":"journal-article","issue":null,"score":1,"title":["Diazoxide Choline Extended-release Tablets in Prader-Willi 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,"}]},{"given":"David A","family":"Stevenson","sequence":"additional","affiliation":[{"name":"Department of Clinical Genetics, Stanford University , Palo Alto, CA 94305 ,"}]},{"given":"Jorge","family":"Mejia-Corletto","sequence":"additional","affiliation":[{"name":"Department of Pediatric Endocrinology, NYU Langone Health , Mineola, NY 11501 ,"}]},{"given":"Mohamad G","family":"Shaikh","sequence":"additional","affiliation":[{"name":"Department of Endocrinology, Royal Hospital for Children , Glasgow G51 4TF ,"}]},{"given":"M Jennifer","family":"Abuzzahab","sequence":"additional","affiliation":[{"name":"Department of Endocrinology, Children's Minnesota , St. Paul, MN 55102 ,"}]},{"given":"Amy","family":"Fleischman","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, Boston Children's Hospital , Boston, MA 02115 ,"}]},{"given":"Virginia","family":"Kimonis","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, University of California, Irvine, and Children’s Hospital of Orange County , Orange, CA 92868 ,"}]},{"given":"Ashley H","family":"Shoemaker","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Vanderbilt University Medical Center , Nashville, TN 37212 ,"}]},{"given":"Anthony","family":"Holland","sequence":"additional","affiliation":[{"name":"Department of Psychiatry, Cambridgeshire & Peterborough NHS Foundation Trust, Fulbourn Hospital , Cambridge CB21 5EF ,"}]},{"given":"Lynne M","family":"Bird","sequence":"additional","affiliation":[{"name":"Department of Genetics, University of California, San Diego/Rady Childrens Hospital , San Diego, CA 92123 ,"}]},{"given":"Kathryn S","family":"Obrynba","sequence":"additional","affiliation":[{"name":"Endocrinology, Nationwide Children's Hospital , Columbus, OH 43205 ,"}]},{"given":"Melissa","family":"Lah","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Indiana University School of Medicine , Indianapolis, IN 46202 ,"}]},{"given":"Elizabeth","family":"Littlejohn","sequence":"additional","affiliation":[{"name":"Department of Pediatric Endocrinology, University of Michigan Health—Sparrow Clinical Research Institute , Lansing, MI 48912 ,"}]},{"given":"Katerina","family":"Harwood","sequence":"additional","affiliation":[{"name":"Department of Pediatric Endocrinology, St. Joseph's University Medical Center , Paterson, NJ 07503 ,"}]},{"given":"Heidi","family":"Shea","sequence":"additional","affiliation":[{"name":"Endocrinology, Research Institute of Dallas , Dallas, TX 75231 ,"}]},{"given":"David","family":"Viskochil","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Utah , Salt Lake City, UT 84108 ,"}]},{"given":"Patricia","family":"Hirano","sequence":"additional","affiliation":[{"name":"Soleno Therapeutics , Redwood City, CA 94065 ,"}]},{"given":"Kristen","family":"Yen","sequence":"additional","affiliation":[{"name":"Soleno Therapeutics , Redwood City, CA 94065 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Extended-release Tablets in Prader-Willi Syndrome: A Randomized, Double-blind, Withdrawal Period Study”. Figshare. Deposited 9 January 2026. 10.6084/m9.figshare.31038112.v2","article-title":null,"journal-title":null,"doi-asserted-by":null},{"DOI":"10.1210/clinem/dgac549","key":"2026011611471055200_dgaf661-B20","year":"2023","issue":"1","author":"Diene","volume":"108","first-page":"4","unstructured":null,"article-title":"Liraglutide for weight management in children and adolescents with Prader-Willi syndrome and obesity","journal-title":"J Clin Endocrinol Metab","doi-asserted-by":"crossref"},{"DOI":"10.1210/clinem/dgad015","key":"2026011611471055200_dgaf661-B21","year":"2023","issue":"7","author":"Roof","volume":"108","first-page":"1696","unstructured":null,"article-title":"Intranasal carbetocin reduces hyperphagia, anxiousness and distress in Prader-Willi syndrome: CARE-PWS phase 3 trial","journal-title":"J Clin Endocrinol Metab","doi-asserted-by":"crossref"},{"DOI":"10.1210/clinem/dgac105","key":"2026011611471055200_dgaf661-B22","year":"2022","issue":"6","author":"Miller","volume":"107","first-page":"e2373","unstructured":null,"article-title":"The efficacy, safety, and pharmacology of a ghrelin o-acyltransferase inhibitor for the treatment of Prader-Willi syndrome","journal-title":"J Clin Endocrinol Metab","doi-asserted-by":"crossref"},{"DOI":"10.3390/ijms24032150","key":"2026011611471055200_dgaf661-B23","year":"2023","issue":"3","author":"Mahmoud","volume":"24","first-page":"2150","unstructured":null,"article-title":"Clinical trials in Prader-Willi syndrome: a review","journal-title":"Int J Mol Sci","doi-asserted-by":"crossref"}],"short-title":[],"article-number":"dgaf661","content-domain":{"domain":[],"crossmark-restriction":false},"original-title":[],"container-title":["The Journal of Clinical Endocrinology & Metabolism"],"reference-count":23,"published-online":{"date-time":null,"timestamp":null,"date-parts":[[2026,1,2]]},"references-count":23,"short-container-title":[],"is-referenced-by-count":0},"unpaywall":{"doi":"10.1210/clinem/dgaf661","is_oa":true,"title":"Diazoxide Choline Extended-release Tablets in Prader-Willi Syndrome: A Randomized, Double-blind, Withdrawal Period Study","updated":"2026-01-19T05:56:36+00:00","published_date":"2025-12-31","best_oa_location":{"url":"https://doi.org/10.1210/clinem/dgaf661","pmh_id":null,"url_for_pdf":null,"url_for_landing_page":"https://doi.org/10.1210/clinem/dgaf661"}}},"created_at":"2026-01-17T09:03:27.421Z","updated_at":"2026-01-20T15:55:50.695Z","tags":[{"paper_doi":"10.1210/clinem/dgaf661","tag_type":"age_group","value":"mixed","value_numeric":null},{"paper_doi":"10.1210/clinem/dgaf661","tag_type":"category","value":"Endocrinology & Metabolism","value_numeric":null},{"paper_doi":"10.1210/clinem/dgaf661","tag_type":"condition","value":"Prader-Willi Syndrome","value_numeric":null},{"paper_doi":"10.1210/clinem/dgaf661","tag_type":"generic","value":"diazoxide choline","value_numeric":null},{"paper_doi":"10.1210/clinem/dgaf661","tag_type":"level_of_evidence","value":"2","value_numeric":null},{"paper_doi":"10.1210/clinem/dgaf661","tag_type":"sample_size","value":"n=77","value_numeric":77},{"paper_doi":"10.1210/clinem/dgaf661","tag_type":"study_design","value":"Randomized Controlled Trial","value_numeric":null},{"paper_doi":"10.1210/clinem/dgaf661","tag_type":"warning","value":"Only abstract-level details were provided (e.g., no dosing specifics or sex distribution).","value_numeric":null},{"paper_doi":"10.1210/clinem/dgaf661","tag_type":"warning","value":"This was a randomized withdrawal design; classified here under randomized controlled trial for simplicity.","value_numeric":null}],"summaries":{"what_studied":"A 16-week, randomized, double-blind withdrawal trial compared once-daily diazoxide choline extended‑release (DCCR) vs placebo in children (≥4 years) and adults with Prader‑Willi syndrome and hyperphagia who had completed prior 13‑week randomized and 2.5–4.5‑year open‑label DCCR studies; the primary endpoint was change in HQ‑CT total score.","key_result":"Among 77 participants (DCCR 38; placebo 39), HQ‑CT worsened less with DCCR than placebo (LS mean change 2.6 vs 7.6; P=0.0022). CGI scores favored DCCR without significant change; placebo gained more weight and increased BMI z‑score more than DCCR (LS mean differences: weight −1.6 kg, 95% CI −3.1 to −0.1; BMI z‑score −0.09, 95% CI −0.17 to −0.01); adverse events were similar, with no serious events in the DCCR arm.","limitations":"Participants had prior DCCR exposure (completed earlier 13‑week randomized and 2.5–4.5‑year open‑label studies). CGI secondary endpoints were not significantly changed, and follow‑up was 16 weeks.","practice_takeaway":"In people ≥4 years with Prader‑Willi syndrome and hyperphagia who had been on DCCR, continuing DCCR for 16 weeks limited worsening of hyperphagia and weight/BMI gain compared with withdrawal, with similar adverse events and no serious events on DCCR."}}],"initialOffset":2,"fetchConfig":{"kind":"tags","tags":[{"tag_type":"condition","value":"Prader-Willi Syndrome"}]},"breadcrumbItems":[{"name":"Home","href":"/"},{"name":"Research Hub","href":"/hub"},{"name":"Conditions","href":"/hub/tag/condition"},{"name":"Prader-Willi Syndrome"}],"hrefConfig":{"basePath":"/hub/papers","webViewQueryParams":{"hub_ctx":"tag","hub_tag_type":"condition","hub_tag_value":"Prader-Willi Syndrome"}}}]]

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