Clinical Trials Alert, April 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 http://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 are changed criteria on older studies, etc – so please read through carefully to see if you might be able to contribute.
It starts with us!  

Ongoing

 

New! Phase 2 Trial to Evaluate Safety & Efficacy of RM-493 in Obese Patients With Prader-Willi Syndrome

 

Rhythm Metabolic, 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.

 

New! 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.  Children with PWS, ages 5-11, are eligible. For more information, visit FPWR
here or clinicaltrials.gov
here.

 

New Sites! New criteria! Phase III Trial of Beloranib in Obese Subjects with PWS (bestPWS)

  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 now open and recruiting. 
In a new development, Canadian citizens can now participate at US sites. 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
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.  

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.  
Got twins who poop? Dr. Shulman is
studying the gut microbiome in individuals with PWS.  Identical and fraternal twins are needed.  Please contact us (
Theresa.strong@fpwr.org) and we’ll put you in touch.  

   

On the Horizon!

 

 

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.
 

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 the coming year.  More information about the program can be found
here.    

Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome

Update:  Enrollment 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 fully completed study and will share them as soon as they are available. For more information about the trial, visit FPWR
here or clinical trials.gov
here.  

 

Recently Completed

 

Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome

Update:  Enrollment 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 fully completed study and will share them as soon as they are available. For more information about the trial, visit FPWR
here or clinical trials.gov
here.  

 

Topics: Admin

Susan Hedstrom

author-image

Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the natural history of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she hosted her first One SMALL Step walk in 2010 and began the development of the One SMALL Step walk program which now raises over $1.5 million a year for PWS research.

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