Intranasal Carbetocin Reduces Hyperphagia, Anxiousness, and Distress in Prader-Willi Syndrome: CARE-PWS Phase 3 Trial
- Enrollment stopped prematurely because of onset of the COVID-19 pandemic
Authors' conclusion
Carbetocin was well tolerated, and the 3.2-mg dose was associated with clinically meaningful improvements in hyperphagia and anxiousness and distress behaviors in participants with PWS.
Study context
- Population
- Participants with Prader-Willi syndrome aged 7 to 18 years; 24 ambulatory clinics at academic medical centersenrollment stopped early (COVID-19)
- Participants
- 130 participants
- Comparison
- Intranasal carbetocin 9.6 mg or 3.2 mg vs placebo, 3 times daily
- Primary endpoint
- Change in hyperphagia (HQ-CT) and obsessive-compulsive symptoms (CY-BOCS), 9.6 mg vs placebo
- Follow-up
- 8-week placebo-controlled period
Methodological detailsdouble-blind, placebo-controlled, phase 3, long-term follow-up, multicentre
- Double-blind
- Placebo-controlled
- Phase 3
- Long-term follow-up
- Multicentre
Primary result
As reported in the abstract
HQ-CT and CY-BOCS, 9.6 mg vs placebo
- Numeric improvements in both HQ-CT and CY-BOCS, not statistically significant
Secondary outcomes
HQ-CT, PADQ and clinical global impression of change (CGI-C), 3.2 mg vs placebo · 8-week placebo-controlled period
- Nominally significant improvements in HQ-CT, PADQ and CGI-C scores with 3.2 mg vs placebo
Long-term follow-up · 56-week long-term follow-up period
- Improvements were sustained in the long-term follow-up period
Safety
Adverse events · 8-week placebo-controlled period
- Most common adverse event: mild to moderate flushing
Original publication
Intranasal Carbetocin Reduces Hyperphagia, Anxiousness, and Distress in Prader-Willi Syndrome: CARE-PWS Phase 3 Trial. J Clin Endocrinol Metab. Published 12 January 2023. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →