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
Developing novel therapeutics for the treatment of rare diseases.