Diazoxide Choline Extended-release Tablets in Prader-Willi Syndrome: A Randomized, Double-blind, Withdrawal Period
Jennifer L Miller1, Nicola Bridges2, Eric I Felner3
1Department of Pediatric Endocrinology, University of Florida College of Medicine, Gainesville, FL 32608, USA.
The Journal of Clinical Endocrinology and Metabolism
|January 3, 2026
Summary
Diazoxide choline extended-release (DCCR) significantly improved hyperphagia in Prader-Willi syndrome patients compared to placebo. DCCR offers a promising therapeutic option for managing this core symptom.
Area of Science:
- Neuroscience
- Genetics
- Metabolic Disorders
Background:
- Prader-Willi syndrome (PWS) is a rare genetic disorder.
- Life-threatening hyperphagia is the hallmark symptom of PWS.
- Current treatment options for PWS hyperphagia are limited.
Purpose of the Study:
- To assess the efficacy and safety of diazoxide choline extended-release (DCCR) for treating hyperphagia in PWS.
- To evaluate DCCR in both adult and pediatric PWS patients (age 4+).
Main Methods:
- A 16-week randomized withdrawal study was conducted.
- Participants received either DCCR or placebo.
- The primary endpoint was the change in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score.
Main Results:
- DCCR treatment resulted in a statistically significant reduction in hyperphagia compared to placebo (P=0.0022).
- Patients on placebo gained more weight and had increased BMI z-scores versus those on DCCR.
- Adverse events were comparable between DCCR and placebo groups, with no serious events in the DCCR arm.
Conclusions:
- Continued DCCR treatment demonstrated superiority over placebo in managing hyperphagia in PWS.
- DCCR shows potential as a meaningful therapeutic intervention for individuals with Prader-Willi syndrome.
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