Acadia Pharmaceuticals is currently enrolling participants in a Phase 3 clinical trial, known as COMPASS-PWS, to assess the safety and efficacy of carbetocin nasal spray in treating hyperphagia in individuals with Prader-Willi Syndrome (PWS). This double-blind study aims to evaluate whether carbetocin, a synthetic analogue of oxytocin, can effectively manage the excessive appetite characteristic of PWS, a condition affecting approximately 1 in 10,000 to 30,000 individuals worldwide.
The COMPASS-PWS trial will involve 170 participants aged 5 to 30 who have been diagnosed with PWS and experience hyperphagia. Participants will be randomized to receive either carbetocin nasal spray or a placebo over a 12-week period. The study design includes five in-person clinic visits: a screening visit, and additional visits at baseline, week 2, week 8, and week 12. Following the initial 12-week study, participants have the option to enroll in an open-label extension study, where all participants will receive carbetocin nasal spray for up to three years.
Carbetocin's Mechanism of Action
Prader-Willi Syndrome is often characterized by a deficiency in oxytocin, a naturally occurring hormone that regulates hunger, anxiety, social behavior, and bonding. Carbetocin, an investigational drug, is designed to bind to oxytocin receptors with greater sensitivity than natural oxytocin, potentially making it more effective in addressing the hormonal imbalance associated with PWS. Carbetocin has been used outside the U.S. in its intravenous form to prevent postpartum hemorrhage, demonstrating a well-established safety profile.
Eligibility and Study Details
Key inclusion criteria for the COMPASS-PWS trial include a confirmed diagnosis of PWS with hyperphagia, an age between 5 and 30 years, and the absence of active upper respiratory infections or a history of brain trauma. Participants must also not have initiated any new food-related interventions within the past month or participated in another treatment study within the past six months. Acadia Pharmaceuticals will review participants' medical histories to determine study eligibility.
Addressing Unmet Needs in PWS
Currently, there are limited effective treatments specifically targeting hyperphagia in PWS. The development of carbetocin nasal spray represents a potential advancement in managing this challenging symptom, which significantly impacts the quality of life for individuals with PWS and their families. The results of the COMPASS-PWS trial could provide valuable insights into the therapeutic potential of carbetocin in addressing hyperphagia and other related symptoms in PWS.
