The University of Alabama at Birmingham is partnering with Children’s of Alabama as a site for the implementation of an international clinical trial — ZEPHYR — aimed at treating children with Prader-Willi syndrome with a new potential medication. 

PWS is a rare endocrine disease that occurs in one in 15,000 individuals, most often children. It causes unrelenting hunger and excessive eating, a root cause of morbidity and mortality in PWS patients.

The ZEPHYR study will evaluate the safety and efficacy of the investigational drug Livoletide as a potential novel treatment for Prader-Willi syndrome. The two-part, randomized, double-blind, placebo-controlled pivotal Phase 2b/3 study will aim to measure the effects of Livoletide on insatiable hunger, body weight, body mass index and body fat mass, as well as levels of safety and tolerance from participants. Additionally, it will look at patients’ weights, as well as administer a questionnaire on excessive appetite, known as hyperphagia, to assess their level of hunger and efforts to search for food while enrolled in the study.

“We are excited that UAB and Children’s of Alabama are a part of a trial that will determine whether we have a viable treatment option for patients living with Prader-Willi syndrome,” said Hussein Abdul-Latif, M.D., pediatric endocrinologist at UAB and Children’s of Alabama and site leader of the study. “Prader-Willi syndrome is a devastating disease for both the patients and caregivers of those living with it, and we are committed to continuing our research in this rare endocrine disease area together.” 

The ZEPHYR study, sponsored by Millendo Therapeutics, is open to participants ages 8 to 65.

PWS is caused by the lack of expression of several genes on chromosome 15, which leads to intellectual disability, short stature, incomplete sexual development and hyperphagia, among other symptoms. Patients are at risk of premature mortality, mainly from obesity-related conditions such as cardiovascular disease or respiratory distress.

Currently, there are no effective or approved treatments for the abnormal eating behaviors associated with PWS. Growth hormones are used for improvement in height, cognition and body composition, but have no effect on appetite and over-eating. The only way to effectively manage hyperphagia, obesity and related complications of PWS is strict control of access to food, creating significant burden for families and caregivers.