Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome: Results From the Randomized, Placebo-Controlled, Phase 3 COMPASS PWS Study.
Elizabeth Roof, Theresa V Strong, Diane E J Stafford et al.
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In 30 seconds
The multicenter, randomized, placebo-controlled phase 3 COMPASS PWS trial assessed carbetocin nasal spray for hyperphagia in people with Prader-Willi syndrome. Over 12 weeks, 175 participants received carbetocin 3.2 mg three times daily or placebo. The primary outcome, change in Hyperphagia Questionnaire for Clinical Trials score at week 12, improved in both groups, with no significant treatment difference: 0.3 (95% confidence interval: -1.8, 2.4; P = 0.79). Carbetocin also did not separate from placebo on secondary or exploratory outcomes.
Key findings
- At week 12, HQ-CT scores changed by -4.8 (0.8) with carbetocin and -5.1 (0.8) with placebo.
- The treatment difference in HQ-CT change was 0.3 (95% confidence interval: -1.8, 2.4; P = 0.79).
- No separation from placebo was observed for secondary or exploratory endpoints.
- The most frequently reported adverse events with carbetocin were headache (n = 6 [7.2%]) and pyrexia (n = 5 [6.0%]).
Why it matters
Hyperphagia is a prominent feature of Prader-Willi syndrome, and carbetocin was studied as a potential treatment targeting oxytocin-related neural pathways. In this confirmatory phase 3 trial, improvement in the primary measure was similar with carbetocin and placebo, and other assessed outcomes also showed no separation. These results help clarify the evidence for this approach in the studied population.
What to keep in mind
The study assessed outcomes over 12 weeks; longer-term effects are not established by the reported results.