PWS Clinical Trials Alert: March 2019

Several NEW PWS trial opportunities are now available. FPWR maintains the latest clinical trial information so that you can stay abreast of trial opportunities. Some trials require in-person visits while others can be completed remotely. Some trials are testing new drugs while others are intervention (non-drug) or natural history studies, like the PWS Weight Study described below.

FPWR does not endorse or recommend any specific study. Our goal is to provide information to members of the PWS community decide whether participation in a clinical trial is right for them and their loved one with PWS. Anyone considering participating in a clinical trial should discuss the matter with his or her physician.

Currently Recruiting

PATH for PWS (ages 5 and up)

PATH for PWS is a 4-year natural history study to help us better understand serious medical events in PWS. The study is 100% survey based and no clinic visits are required. The study needs 175 more participants ages 12 and up.  Receive an Amazon gift card for completing surveys every 6 months. Learn More >>

Carbetocin in PWS (ages 7 - 18) -- WATCH THE RECORDED WEBINAR 

This Phase 3 study of Carbetocin in PWS is enrolling patients ages 7 to 18 at 23 sites across the US and Canada. This is an 8 week study, with the option of an open label extension, measuring the efficacy, safety and tolerability of Carbetocin for the treatment of hyperphagia and PWS related behaviors. 
Watch the Webinar >>

DCCR for the Treatment of Hyperphagia in PWS (ages 4 and up) -- WATCH THE RECORDED WEBINAR 

12 study sites are now open for Soleno Therapuetics' Phase 3 study of DCCR in PWS. This study is recruiting patients ages 4 years and older for a 15 week study measuring the efficacy of DCCR for treating hyperphagia. Additional sites are expected to open soon. Learn more >>
Watch the Webinar >>

Cannabidiol in PWS (ages 8 - 17) -- WATCH THE RECORDED WEBINAR 

Insys Therapeutics is conducting a Phase 2 study of Cannabidiol (CDB) to evaluate whether the use of CBD, provided as an oral solution, can help control hyperphagia and compulsive behaviors. The first of several study sites is now open in Tacoma, WA and enrolling patients 8 to 17 years old. Additional sites are opening soon (San Diego, Iowa City, Tuscon, Kansas City, Tulsa, Memphis, Baltimore, Boston). Learn more >>
Watch the Webinar >>

Intranasal Oxytocin for the Treatment of Hyperphagia in PWS (ages 5 - 17)

This is a randomized, double blind Phase 2 study of intranasal oxytocin in PWS. The study is now enrolling patients ages 5-17 years old. Travel to the Montefiore Medical Center, Bronx, NY is required for 5 on-site visits in addition to 2 phone call visits. Learn more >>

Mindfulness-Based Intervention for Temper Outbursts (ages 13 - 30)

The study is looking for individuals with and without PWS ages 13 to 30 years who have either a mild intellectual disability or typical IQ. This study will be conducted remotely via phone, email and video-conference and is, therefore, open to families both within and outside of Australia. Learn more >>

Oxytocin and the Autonomic Nervous System in PWS (ages 13-30)

Patients ages 13-30 are needed to participate in a study aiming to understand the nature of the oxytocin abnormality in PWS. The study will be conducted over a 2 hour visit at either the University of Sydney in Camperdown, NSW or the Royal Children’s Hospital in Parkville, Victoria. Learn more >>

PRETEND Remote Play-Based intervention Study (ages 6 - 9) - COMING TO DC

A special call-out to PWS families in the Washington DC area! The Dimitropoulos Team is visiting the area this spring, and will be actively enrolling for the remaining spots in the PRETEND study. This study is focused on optimizing development in children with PWS and is recruiting families of children with PWS ages 6-9 years old. This study includes both direct play-based intervention for children over a remote video-conferencing platform to increase children's play skills, emotional regulation, and cognitive and behavioral flexibility. The study takes 8 weeks, and only requires TWO in person visits in your area - once at the beginning of the study and once at the end. We are also recruiting families of children with PWS ages 3-12, as well as siblings, for a one-time visit eye-tracking study.  For questions on either study, you can contact the research team via email or by phone at 216-368-0112.

Eye Tracking - Developing Objective Biomarkers of Hyperphagia (ages 3 - 12)

This study requires a single in-person visit. Eye tracking technology will be used to measure how children process images of food vs. objects. The aim of this study to to determine if eye tracking can help us better understand and measure hyperphagia objectively.  For this study, both children with PWS and neurotypically developing peers are needed, ages 3-12. This study is being done in collaboration with the team at Vanderbilt University. Learn more >>


Clinical Trials Overview

A multitude of factors come in to play when determining whether to participate in a clinical trial. To help make it easier to see which trials you may be right for you and your family, we have generated an easy to read matrix which compares common variables such as age and BMI requirements, study sites and estimated start dates. Click here to see the full PWS Clinical Trials matrix.

Find a Trial Near You

Use our interactive map of clinical trials!


Donation of 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. For details, click here

Post-Mortem Brain Donation.

Organ donation is a highly personal decision that has the power to accelerate research and transform the lives of countless families affected by PWS. You can make a tremendous difference in research into Prader-Willi syndrome by participating in this program. In the event of a death, contact the 24-hour hotline number: 1-877-333-0999 for immediate assistance. Learn more >>

While we are pleased to share with you these opportunities for participating in PWS clinical trials, FPWR does not endorse or recommend any specific trial. 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.

Visit PWS Clinical Trials Page

Topics: Research

Susan Hedstrom


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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