France-based Alizé Pharma has started a Phase II clinical trial of AZP-531, its unacylated ghrelin analog, to treat patients with Prader-Willi syndrome, a rare genetic metabolic syndrome.
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The randomized, double blind and placebo controlled trial, which will be conducted in several European countries, is designed to assess the safety and effects of a two-week treatment with AZP-531 on food-related behavior and on weight in about 40 patients with Prader-Willi syndrome.
The company said that sites are open for recruitment in France and Spain with results scheduled to be available by mid-2016.
Prader-Willi syndrome is characterized by hyperphagia, an excessive eating behaviour and severe obesity. By inhibiting the effects of increased acylated ghrelin blood levels in these patients, AZP-531 helps in improving hyperphagia.
AZP-531 is also expected to decrease weight and improve glucose control, a key additional benefit as 25% of adult Prader-Willi patients have type 2 diabetes.
Hospital of Toulouse pediatric endocrinologist, coordinator of the Reference Center for Prader-Willi in France and principal investigator of the trial Maïthé Tauber said: "AZP-531, an unacylated ghrelin analog, could constitute an attractive therapeutic approach by targeting a key underlying endocrine defect of the syndrome."
The new Phase II trial is the fourth AZP-531 clinical trial to be launched by the company, which has already completed two Phase I trials in healthy volunteers and obese subjects.
Data from these trials showed that AZP-531 was well tolerated, with improved glucose control and decreased weight in obese subjects over a two-week treatment period.
The company has also started a Phase Ib trial in type 2 diabetes in 2014 with results expected in the second half of 2015.