Pilot Study of DCCR in PWS Patients (PC025)

Clinical Trial of DCCR for PWS

A Phase II study, single-center, randomized withdrawal study of DCCR in obese PWS subjects has been completed. This study evaluated the safety and preliminary efficacy of DCCR treatment in 13 subjects with genetically-confirmed PWS who were ages 10 – 21 years of age.

During the 10-week, open-label treatment phase, a statistically significant reduction in hyperphagia from baseline to the end of the open-label treatment phase was observed. In addition, greater improvement in hyperphagia from baseline was observed in those subjects with moderate to severe hyperphagia who received DCCR doses of 4.2 mg/kg. There was a statistically significant improvement in the number of subjects reporting one or more aggressive and destructive behaviors. A mean decrease in body fat mass and increases in lean body mass and lean body mass / fat mass ratio were seen. These changes were associated with a statistically significant reduction in waist circumference, consistent with the loss of visceral fat. Statistically significant reductions from Baseline in LDL cholesterol, and non-HDL cholesterol were observed. The change in triglycerides, while marked, did not reach statistical significance.

PC025 Poster Presentation at the 10th International Meeting of Pediatric Endocrinology