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Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome: Results From the Randomized,
Elizabeth Roof1, Theresa V Strong2, Diane E J Stafford3
1Vanderbilt University, Nashville, Tennessee.
Purpose:
Prader-Willi syndrome (PWS) is a rare genetic disorder characterized by endocrine and neuropsychiatric problems including hyperphagia, anxiousness, and distress. The oxytocinergic system represents one of the key neural circuits that is dysfunctional in PWS, making it an attractive therapeutic target. The oxytocin analog carbetocin has a longer half-life and greater receptor selectivity than oxytocin and showed therapeutic potential in the phase 3 CARE-PWS study (ClinicalTrials.gov NCT03649477) based on nominally significant improvements in hyperphagia and anxiousness with the 3 times daily (TID) 3.2-mg dose of intranasal carbetocin over placebo. The phase 3 placebo-controlled COMPASS PWS study was conducted to confirm the potential benefit observed with carbetocin in the CARE-PWS study.
Methods:
In the 12-week COMPASS PWS study (ClinicalTrials.gov NCT06173531), 175 participants were randomized 1:1 to carbetocin 3.2 mg TID nasal spray (n = 85) and placebo (n = 90). The primary efficacy endpoint was the change from baseline at week 12 in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score. Secondary efficacy endpoints were the change in the Clinical Global Impression-Severity (CGI-S) score for PWS, and in the CGI-S for hyperphagia in PWS score from baseline at week 12, the CGI-Change for PWS score at week 12, and the percentage of participants with a treatment response (defined as improvement from baseline ≥8 points) based on the HQ-CT at week 12. Exploratory efficacy endpoints included the change from baseline at week 12 in scores for the PWS Anxiousness and Distress Behaviors Questionnaire.
Findings:
The least squares mean change (standard error) from baseline at week 12 in the HQ-CT was -4.8 (0.8) and -5.1 (0.8) in the carbetocin and placebo groups, respectively; the treatment difference of 0.3 (95% confidence interval: -1.8, 2.4; P = 0.79) was not statistically significant. There was no separation between carbetocin and placebo for any secondary or exploratory endpoint. The most frequently reported treatment-emergent adverse events in the carbetocin-treated participants were headache (n = 6 [7.2%]) and pyrexia (n = 5 [6.0%]).
Implications:
Carbetocin nasal spray did not demonstrate efficacy compared with placebo for hyperphagia in PWS in the 12-week COMPASS PWS study.
