US-based pharmaceutical firm Essentialis has dosed the first patient in clinical study PC025, which is designed to evaluate the safety and efficacy of diazoxide choline controlled release tablets (DCCR) in patients with obese Prader-Willi syndrome (PWS).
A total of 12 PWS patients between ten and 20 years old will be enrolled in the single-centre, randomised withdrawal trial.
Patients will be titrated through five doses of DCCR over ten weeks of open label treatment followed by a four week double-blind, placebo-controlled, randomised withdrawal phase during which half of the patients in the study will be randomised to continue on DCCR and half will be randomized to placebo.
The trial’s endpoints include safety measures, and changes in hyperphagia, weight, fat mass and resting energy expenditure.
The company said that these endpoints will be measured both during the open label titration phase and the double-blind, placebo-controlled, randomised withdrawal phase.
Essentialis president and chief scientific officer Neil Cowen said: “The speed with which patients for this study are being recruited and qualified is a testament to the hard work of Dr Virginia Kimonis’ team and the staff of the ICTS at the University of California, Irvine, and the assistance and encouragement of the Prader-Willi California Foundation, The Foundation for Prader-Willi Research, Children’s Hospital of Orange County and the commitment of parents and caregivers of PWS patients.”
The company said that open label data from this trial is available effective July and intends to release the double-blind, placebo-controlled results in the fourth quarter of 2014.
DCCR is a proprietary crystalline salt of diazoxide formulated as a controlled-release, once-a-day tablet and it is being developed to treat Prader-Willi syndrome and hypothalamic obesity.
The company said that DCCR is covered by multiple issued US and granted EU and Japanese patents, which provide composition of matter protection until 2028.