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PWS Clinical Trials Alert

The latest news and opportunities for PWS clinical trials taking place to help us understand Prader-Willi syndrome and investigate new treatments.

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.

Drug Trials Recruiting or Coming Soon

LIGHT THERAPY (AGES 6 -18) - NEW - NO TRAVEL REQUIRED

This study is evaluating the effectiveness of bright light therapy in reducing daytime sleepiness in PWS. This study is 100% remote and does not require any travel. All study visits will be conducted via teleconference. Learn more >>

VNS4PWS FOR DISRUPTIVE BEHAVIOR (AGES 10-40) - 3 SITES NOW OPEN (CA, NY, TX)

Does your loved one struggle with disruptive behavior and temper outbursts? The VNS4PWS trial will enroll people with PWS ages 10-40 with a history of temper outbursts over the past 6 months. Learn more >>

CARBETOCIN FOR HYPERPHAGIA IN PWS (AGES 5 - 30) - 3 SITES NOW OPEN (IA, MO, NY, TN)

In this Phase 3 study, carbetocin nasal spray or placebo will be inhaled through the nose three times each day. This study will last approximately 19 weeks and will involve up to 5 in-person visits to the study center. Upon completion of the study treatments, participants are invited to join a long-term extension study in which all participants will receive carbetocin nasal spray for up to 36 months. Learn more >>

NNZ-2591 FOR THE POTENTIAL TREATMENT OF PWS BEHAVIORS (AGES 4 -12) - 3 SITES NOW OPEN (GA, MD, PA)

This Phase 2 study will evaluate the safety and effectiveness of NNZ-2591 (an oral solution) in treating PWS behaviors. The study is currently enrolling children with PWS ages 8 - 12. Enrollment for children ages 4 - 7 will open soon. This 13-week study requires 5 in-person study visits. Additional trail sites will be coming soon. Learn more >>

COMING SOON - PITOLISANT FOR EXCESSIVE DAYTIME SLEEPINESS IN PWS

This Phase 3 study will be available for people with PWS ages 6+ who struggle with excessive daytime sleepiness.


 

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.

WellCAST: Supporting Wellbeing of Caregivers Via Telehealth

Project WellCAST is a NIH-funded clinical trial that aims to support caregiver well-being through supportive therapy, resources, and family friendly treatment. This research study will include up to 1,000 caregivers of children with a variety of neurogenetic syndromes associated with intellectual disability, including (but not limited to) Angelman syndrome, Down syndrome, Dup15q syndrome, fragile X syndrome, Prader Willi syndrome, and Williams syndromes. Learn more >>

Behavioral Training for Caregivers of Children with PWS

This 15-week training program is 100% remote. Caregivers will participate in an online program with live and recorded sessions. Participants must have a child with PWS aged 3-12 who engages in at least one disruptive behavior such as tantrums, hitting, picking at skin, or refusing to comply with necessary tasks. Learn more >>


 

Clinical Trials Overview

A multitude of factors come into play when determining whether to participate in a clinical trial. To help make it easier to see which trials may be right for you and your family, we have generated an easy-to-read matrix that 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!

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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.

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

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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 future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.