JUNE 2015.

There are several new and ongoing opportunities for PWS families to participate in clinical studies. For more information on the opportunities below, as well as others, please visit https://www.fpwr.org/category/clinical-trials or clinicaltrials.gov. We encourage members of the PWS community to seek out information that will help them decide whether participation is right for them. Please contact the study coordinators directly for important details about each study and to answer any questions you may have. Some trials may have funds available to support travel to study sites.

In addition to new studies, there may be changed criteria for older studies – so please read through carefully to see if you might be able to contribute. It starts with us!


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/

Tissues Needed
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.


tDCS modulation of hyperphagia in PWS
A recently completed pilot study shows promise that a weak, non-invasive form of brain stimulation called transcranial direct current stimulation (tDCS) may reduce food cravings and overeating in PWS. A larger follow up trial is scheduled to start soon and will help determine if the use of tDCS brain stimulation would be an effective, noninvasive, inexpensive treatment to control food cravings and overeating in adults with PWS. This trial is funded, in part, by FPWR’s research grant program. Click here for more information on this project.  Look for this study to open in Summer 2015.
Alize Pharma announces plans to test AZP-531 in PWS
Alize Pharma has developed a drug targeting ghrelin, the “hunger hormone” abnormally elevated in PWS.  AZP-531, an unacylated ghrelin analog, could potentially reduce hyperphagia in PWS.  The company recently announced that the drug was well tolerated and resulted in weight loss in a 14 day, Phase I trial of healthy volunteers and obese individuals.  Alize plans to test AZP-531 in individuals with PWS in 2015.  More information about the program can be found here.
RECENTLY COMPLETEDWe will keep you informed as study results are released!

Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
Update:  Study Completed, Thank You PWS Community!!
Results from the first phase of the study were recently reported.  We look forward to learning the results of the completed study and will share them as soon as they are available. For more information about the trial, visit FPWR.

 Oxytocin Trial in Prader-Willi Syndrome
Update: Enrollment completed – Thank you PWS Community!!
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.  Children with PWS, ages 5-11, are eligible. For more information, visit FPWR here or clinicaltrials.gov here.

 Phase III Trial of Beloranib in Obese Subjects with PWS (bestPWS)
Update: Enrollment completed – Thank you PWS Community!!
Zafgen is conducting a Phase III trial of its drug, Beloranib, in adolescents and adults with PWS.  The study will determine whether this drug helps people with PWS burn fat more efficiently and feel less hungry. There are fourteen study sites across the US have been opened and are active. Zafgen will assist families with travel expenses, and all participants who complete the first six months of the study, including those who initially receive placebo, will have the opportunity to receive beloranib for an additional six months if they so choose. The study is for adolescents and adults (12 and over) with a confirmed PWS diagnosis and who are overweight or obese.  For more information, visit FPWR here or clinicaltrials.gov.