Diazoxide Choline Extended-Release Tablet in People With Prader-Willi Syndrome: A Double-Blind, Placebo-Controlled Trial
Authors' conclusion
DCCR did not significantly improve hyperphagia in the primary analysis but did in participants with severe baseline hyperphagia and in the pre-COVID analysis. DCCR treatment was associated with significant improvements in body composition and clinician-reported outcomes.
Study context
- Population
- Participants with Prader-Willi syndrome aged 4 years and older with hyperphagia (DESTINY prader-Willi syndrome (PWS))randomized 2:1
- Participants
- 127 participants
- Comparison
- Diazoxide choline extended-release tablet vs placebo
- Primary endpoint
- Change from baseline in hyperphagia (Hyperphagia Questionnaire for Clinical Trials, HQ-CT)
- Follow-up
- 13 weeks
Methodological detailsdouble-blind, placebo-controlled, phase 3, randomized 2:1, 13-week
- Double-blind
- Placebo-controlled
- Phase 3
- Randomized 2:1
- 13-week
Primary result
As reported in the abstract
Change in HQ-CT
DCCR
−5.94
least-square mean · SE 0.879
Placebo
−4.27
least-square mean · SE 1.145
P = 0.198
Secondary outcomes
Least-square mean
| Outcome | DCCR | Placebo | P value |
|---|---|---|---|
| Change in HQ-CT, participants with severe hyperphagia | −9.67 (SE 1.429) | −4.26 (SE 1.896) | P = 0.012 |
- Secondary end points: CGI-I and fat mass · improved
- P = 0.029
- Secondary end points: CGI-I and fat mass · improved
- P = 0.023
- Pre-COVID analysis: Hyperphagia Questionnaire for Clinical Trials (HQ-CT), CGI-I, Caregiver Global Impression of Change, fat mass · improved · analysis of results generated pre-COVID
- P = 0.037
- Pre-COVID analysis: HQ-CT, CGI-I, Caregiver Global Impression of Change, fat mass · improved · analysis of results generated pre-COVID
- P = 0.015
- Pre-COVID analysis: HQ-CT, CGI-I, Caregiver Global Impression of Change, fat mass · improved · analysis of results generated pre-COVID
- P = 0.031
- Pre-COVID analysis: HQ-CT, CGI-I, Caregiver Global Impression of Change, fat mass · improved · analysis of results generated pre-COVID
- P = 0.003
Secondary end points: CGI-I and fat mass
- Two of 3 secondary end points improved
Safety
Treatment-emergent adverse events
Treatment-emergent adverse events
- DCCR generally well tolerated; difference not significant
Original publication
Diazoxide Choline Extended-Release Tablet in People With Prader-Willi Syndrome: A Double-Blind, Placebo-Controlled Trial. J Clin Endocrinol Metab. Published 14 January 2023. doi:10.
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