PWS Clinical Trials
There are several new opportunities for PWS families to participate in clinical studies. You can learn more about the studies below, and others, at fpwr.org or at clinicaltrials.gov. We encourage you to contact the study coordinator directly to get important details about the study, to help you decide whether participation is right for you. Funds may be available to support travel to institutions for the studies.
Oxytocin Trial in Prader-Willi Syndrome
Individuals with Prader-Willi syndrome (PWS) have a reduced number of oxytocin-producing neurons and decreased oxytocin receptor function. This Phase 1 study will determine if oxytocin (OT) administration improves some of the aspects of PWS, including insatiable appetite and problems with social behaviors. There are (3) study sites – California, Florida, and Kansas (ages 5-11). Florida is currently recruiting, with Kansas and California expected to open the study by the summer. See: http://www.fpwr.org/oxytocin-trial-in-prader-willi-syndrome/
Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
In cooperation with University of California Irvine, Essentialis Therapeutics is conducting a Phase 1 clinical trial testing the impact of diazoxide choline on hyperphagia, energy expenditure, and weight in individuals with PWS. This study is taking place in California (ages 10-20). See: http://www.fpwr.org/clinical-study-of-diazoxide-choline-controlled-release-tablet-dccr-in-patients-with-prader-willi-syndrome/
Treatment of Hyperphagia Behavioral Symptoms in Children and Adults Diagnosed With Prader-Willi Syndrome
Ferring Pharmaceuticals is conducting a Phase II trial to evaluate the safety and effectiveness of an intranasal oxytocin analogue in treating hyperphagia in children and young adults with Prader-Willi syndrome. Two study sites are still enrolling – New York and in Florida (ages 10-18). See: http://www.fpwr.org/carbetocin-a-selective-oxytocin-agonist-in-the-treatment-of-pws/
Prader-Willi Syndrome Macronutrient Study
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 a broad array of 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 to a greater degree and for longer duration than 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 in North Carolina and in Alberta Canada (ages 5-17). See: http://www.fpwr.org/prader-willi-syndrome-macronutrient-study/
For additional information on clinical trials, visit the fpwr.org website.