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. Details of recruiting studies can be found below.

Clinical Trials Recruiting Now

CSTI-500 for Obesity

This is an approximately 12-week trial of CSTI-500, an oral investigational medication designed to affect serotonin, dopamine, and norepinephrine signaling in the brain, neurotransmitters that play important roles in mood regulation, impulse control, emotional regulation, and hunger. Study participants must be between the ages of 13 and 50 and have PWS. The study includes regular clinic visits and study assessments at Vanderbilt University. Learn more about this study>>

RM-718 for Obesity & Hyperphagia

RM-718 is an open-label study in which participants will receive a weekly injection of RM-718 for 26 weeks. Participants must be between the ages of 12 and 65 and have a body mass index (BMI) of 30 or more for adults, or a BMI in the 95th percentile for those under 18. Eligible participants who complete the study will have the option to continue receiving RM-718 during a Long-Term Extension Period. Learn more about this study>>

Pitolisant for Excessive Daytime Sleepiness

The Phase 3 TEMPO study is enrolling participants ages 6+ who struggle with excessive daytime sleepiness. 30+ sites are now available. The new TEMPO at-home site requires NO TRAVEL with flexible scheduling.  For more information, visit www.tempopwsstudy.com

Tirzepatide for Obesity

This is a 48-week trial of Tirzepatide that includes 6 in-person study visits, 4 telehealth visits, and an additional visit 8 weeks after stopping the medication. Study participants must be between the ages of 12 and 26 and have a BMI of 30 or greater. Participants will receive a $50 gift card per visit, as well as Tirzepatide for 1 year. Learn more about this study>>

Online Study Participation Opportunities

Global PWS Registry

The Global PWS Registry is a powerful tool for the PWS community to advance areas of unmet need, standards of care, and new therapies. Data from the registry is shared back to the PWS community and is also used by researchers and scientists. Visit the registry at www.pwsregistry.org.

Repetitive Verbal Behavior Training Program

Dr. Kasey Bedard would like to learn more about repetitive verbal behavior in PWS. If your loved one asks repetitive questions or makes repetitive statements, please consider completing her 30-minute survey. Participants will be entered for a chance to win 1 of 10 $100 gift cards. Take the Survey >>

Assessing Creativity & Play in PWS study  

Dr. Anastasia Dimitropoulos's research lab is currently investigating the long-term effects of play intervention programs for children with Prader-Willi Syndrome. Participation in this study would involve a one-time virtual visit with your child. This visit is estimated to take approximately 60-90 minutes. Compensation is provided for participation. 

Email: neurodevelopmentresearchlab@gmail.com
Phone number: 216-368-0112
Website: https://caslabs.case.edu/dimitropouloslab/

Bringing PRETEND to You study

This is a continuation of previous PRETEND projects (the Play-based Remote Enrichment To ENhance Development program). Our lab has spent multiple years building, testing, and refining the PRETEND program, which shows evidence of improving play & related socioemotional skills for children with PWS. We are now looking to train other adults and providers to deliver the PRETEND Program to increase access to this effective treatment program. 

Email: neurodevelopmentresearchlab@gmail.com
Phone number: 216-368-0112
Website: https://caslabs.case.edu/dimitropouloslab/

GoCarer Caregiver Survey

The GoCarer Study aims to better understand caregiver burden and stress in families affected by Prader-Willi syndrome (PWS). Caregivers must be 18 or older and caring for a person with PWS between the ages of 2 and 25. Participants will complete two sets of online surveys and five educational modules as part of the study. The study is led by Dr. Andrea Haqq at the University of Alberta. Learn More >>

BRAVE PWS Caregiver Study

The BRAVE PWS Caregiver Study collects real-world health information over time to better understand the daily experiences of individuals with Prader-Willi syndrome (PWS) and their caregivers. Caregivers of a person with PWS age 4 or older can participate by completing short monthly surveys and health tracking. The study is conducted by Folia Health and sponsored by Soleno, a Neurocrine Biosciences company. Learn More >>

Donation of Tissues Needed

Scientists need samples to study PWS – without your contributions, these studies can’t make progress!

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.

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.