Phase 2 Trial to Evaluate Safety & Efficacy of RM-493 in Obese Patients with Prader-Willi Syndrome
Rhythm Pharmaceuticals, Inc. has opened a Phase 2 study of RM-493 at the University of Florida. The purpose of this study is to evaluate the effects of a once daily injectable formulation in obese PWS individuals ages 16-65. The study will measure drug safety and tolerability, as well as weight loss and hyperphagia-related behavior. For more information, visit FPWR here
or clinicaltrials.gov here
. Or, you can watch the webinar
in which Dr. Keith Gottesdiener from Rhythm and Dr. Jennifer Miller from the University of Florida provide details about this study.
Look for new study sites, coming soon!
Dr. Kate Woodcock is studying “task switching” difficulties in PWS, with the goal of developing tools and techniques to help those with PWS cope with unexpected change. Her group is recruiting for three studies related to this challenge. The first study, for children with PWS ages 5 years and up, seeks to understand how ‘resistance to change’ develops. The second study, PREDICTORS (Parent Resources for Decreasing the Incidence of Change Triggered Temper Outbursts) is seeking parents and caregivers of children with PWS (age 7-16) to test and optimize strategies to reduce temper outbursts. Finally, the TASTER project (Training Attention Switching for Temper Episode Reduction) is developing a computer software training program to improve task switching abilities. Dr. Woodcock is located in Ireland, but the studies can be completed remotely by phone or online. More details about each of these studies can be found on FPWR’s website, here
Prader-Willi Syndrome Macronutrient Study – Alberta and Duke
The overall objective of this study is to explore how different foods regulate hyperphagia, satiety, food intake and weight gain in PWS. Using a cutting edge metabolomics approach, researchers will measure hormones, cytokines, amino acids and fatty acid metabolites prior to and 48 hours after either a low carbohydrate or low fat diet. The study will also ask whether a low carbohydrate diet suppresses levels of the hunger hormone ghrelin and increases appetite-suppressing hormones compared to a low fat diet. Information obtained from this study will be used to design evidence-based diet plans for children with PWS. There are (2) study site locations – Duke University in North Carolina and University of Alberta, Alberta, Canada (ages 5-17). For more information, visit FPWR here
or clinicaltrials.gov here
Diagnosis of Central Adrenal Insufficiency in Patients with Prader-Willi Syndrome
Central adrenal insufficiency (CAI) is a potentially serious condition that may be more common in the PWS population than in the normal population. Investigators at Nationwide Children’s Hospital in Columbus, OH, are studying how best to diagnose CAI in PWS, using two different tests. For more information, visit FPWR here
or clinicaltrials.gov here.
Attention Fathers – We Need You!!
Are you a father of a child with Prader-Willi syndrome? The University of Newcastle is interested in your experiences of well-being, coping styles, and access to support, as well as your level of growth since your child’s diagnosis. If you want more information, or would like to complete the questionnaire, please follow this link: http://www.findlab.net.au/are-you-a-father-of-a-child-with-a-genetic-syndrome/
Scientists need samples to study PWS – without your contributions, these studies can’t make progress!Got baby teeth?
Share them with Dr. Reiter. Fresh baby teeth are needed, particularly from those with PWS by UPD. You must have a kit ahead of time. More details here
Got cord blood? Consider sending a sample of cord blood from your child with PWS to Dr. Lalande, see details here.