Diazoxide Choline Extended-release Tablets in Prader-Willi Syndrome: A Randomized, Double-blind, Withdrawal Period Study
Authors' conclusion
Continued DCCR treatment was superior to placebo for hyperphagia. DCCR appears to offer meaningful therapeutic benefits for people with Prader-Willi syndrome.
Study context
- Population
- Children and adults with Prader-Willi syndrome and hyperphagia who had completed prior randomized (13-week DCCR or placebo) and open-label (2.5–4.5 years DCCR) studiesrandomized 1:1
- Participants
- 77 children
- Comparison
- Continued once-daily DCCR vs placebo
- DCCRn = 38
- Placebon = 39reference
- Primary endpoint
- Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score change from baseline
- Follow-up
- 16 weeks
Methodological detailsrandomized withdrawal, double-blind, placebo-controlled
- Randomized withdrawal
- Double-blind
- Placebo-controlled
Primary result
As reported in the abstract
HQ-CT total score change from baseline · week 16
DCCR
2.6
Least square (LS) mean · SE 1.12
Placebo
7.6
LS mean · SE 1.09
P = 0.0022
Secondary outcomes
| Outcome | LS mean weight difference (kg) (95% CI) | |
|---|---|---|
| Weight (exploratory)Placebo gained more weight | −1.6 (−3.1 to −0.1) | |
| −4−202Difference, kg (linear scale) |
| Outcome | LS mean z-score difference (CI) |
|---|---|
| BMI z-score (exploratory)Placebo increased BMI z-score more | −0.09 (−0.17 to −0.01) |
Clinical Global Impression of Severity (CGI-S) and CGI-I
- CGI scores favored DCCR but were not significantly changed
Safety
- Serious adverse events in DCCR
- 0
- Adverse events similar with both treatments
Original publication
Diazoxide Choline Extended-release Tablets in Prader-Willi Syndrome: A Randomized, Double-blind, Withdrawal Period Study. J Clin Endocrinol Metab. Published 2 January 2026. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →