Diazoxide Choline Extended-Release Tablets in Prader-Willi Syndrome: A Randomized, Double-Blind, Withdrawal Period Study
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Authors
Miller J.L.
Bridges N.
Felner E.I.
Salehi P.
Yanovski J.A.
Stevenson D.A.
Mejia-Corletto J.
Shaikh M.G.
Abuzzahab J.
Fleischman A.
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2026
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CONTEXT: The hallmark condition of Prader-Willi syndrome, a rare, genetic neurobehavioral/metabolic disorder is life-threatening hyperphagia. OBJECTIVE(S): 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. METHOD(S): 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. RESULT(S): 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. CONCLUSION(S): Continued DCCR treatment was superior to placebo for hyperphagia. DCCR appears to offer meaningful therapeutic benefits for people with Prader-Willi syndrome.Copyright © The Author(s) 2026. Published by Oxford University Press on behalf of the Endocrine Society. All rights reserved. For commercial re-use, please contact reprints@oup.com for reprints and translation rights for reprints. All other permissions can be obtained through our RightsLink service via the Permissions link on the article page on our site-for further information please contact journals.per
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The Journal of clinical endocrinology and metabolism