Diazoxide choline extended‐release tablet in people with Prader‐Willi syndrome: results from long‐term open‐label study
Authors' conclusion
DCCR administration to people with PWS was well tolerated and associated with broad-ranging improvements in the syndrome. Sustained administration of DCCR has the potential to reduce disease severity and the burden of care for families.
Study context
- Population
- People with Prader-Willi syndrome (PWS) aged ≥4 years enrolled in the DESTINY PWS phase 3 study and/or its open-label extension
- Participants
- 125 people
- Outcome
- Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score
- Follow-up
- Up to 52 weeks
Methodological detailslong-term, single-group
- Long-term
- Single-group
Main finding
As reported in the abstract
Change in HQ-CT score
DCCR
−9.9
mean · SE 0.77
P < 0.0001
Further findings
Aggression, anxiety and compulsivity
- Improvements in aggression, anxiety and compulsivity (all p < 0.0001)
Hormonal measures
- Reductions in leptin, insulin and insulin resistance; significant increase in adiponectin (all p < 0.004)
Lean body mass
- Lean body mass increased (p < 0.0001)
Disease severity
- Disease severity reduced as assessed by clinician and caregiver (both p < 0.0001)
Safety
Adverse events
- Discontinuation due to AEs
- 7.2%
- Common treatment-emergent AEs: hypertrichosis, peripheral edema, hyperglycemia
Original publication
Diazoxide choline extended‐release tablet in people with Prader‐Willi syndrome: results from long‐term open‐label study. Obesity (Silver Spring). Published 2 November 2023. doi:10.
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