Essentialis announced today the dosing of the first patient in clinical study PC025, which is designed to evaluate the safety and efficacy of diazoxide choline controlled release tablets (DCCR) in obese Prader-Willi syndrome patients.
"Although we are announcing today the dosing of the first patient, fully one third of the patients for the study have already been qualified and are scheduled for dosing and another third are currently scheduled for screening. 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," said Dr. Neil M. Cowen, President and Chief Scientific Officer of Essentialis. "We are looking forward to open label data from this study being available starting in July and double-blind, placebo-controlled results in Q4."
Trial Design Clinical trial PC025 (www.clinicaltrials.gov, NCT02034071) is a single-center, randomized withdrawal study of DCCR in genetically confirmed, obese PWS patients. Twelve PWS patients between 10 and 20 years old will be enrolled in the study. Patients will be titrated through 5 doses of DCCR over 10 weeks of open label treatment followed by a 4 week double-blind, placebo-controlled, randomized withdrawal phase during which half of the patients in the study will be randomized to continue on DCCR and half will be randomized to placebo. Endpoints include safety measures, and changes in hyperphagia, weight, fat mass and resting energy expenditure. These endpoints will be measured both during the open label titration phase and the double-blind, placebo-controlled, randomized withdrawal phase.