Clinical Trials Pave The Way For PWS Treatments

PWS clinical trials are taking place around the world to help us understand Prader-Willi syndrome and investigate new treatments; please see the list below for active and pending clinical trials. Click on the link at the left of each column for additional information about each of the clinical trials that are active or are about to start. We encourage you to learn more about these clinical trials, talk with your physician about potential risks and benefits, and ask questions to help you decide if enrolling in a clinical trial is right for you or your loved one with PWS.

You can watch a video on clinical trials here or by clicking on the embed below. To learn more about clinical trials and the questions you might want to ask as a potential participant, check out this page on the NIH website

Your participation is key to moving potential therapies forward. If you or someone you care for has PWS, learn about clinical trials by subscribing to FPWR's Clinical Trial Alerts — just fill out the form below!

Sign Up For PWS Clinical Trial Alerts

PWS Clinical Trials In Progress / Starting Soon

Drug / link Phase Sponsor Status Primary Target Ages BMI # Patients Length Site Locations
GLWL-01 Phase 2 GLWL Research Recruiting Hyperphagia 16 - 65 27 - 60 kg/m2 34 18w

San Diego, CA   Gainesville, FL Nashville, TN  Cleveland, OH Edmonton, AB 
Montreal, QC

Cannabidiol (CBD) Oral Solution Phase 2 Insys Therapeutics Not yet recruiting,
starts June 2018
Hyperphagia 8 - 17 n/a 66 12w TBD
Oxytocin Phase 2 Montefiore Medical Center Not yet recruiting,
starts Spring 2018
Hyperphagia, Repetitive Behavior 5 - 17 n/a 50 8w Bronx, NY
DCCR Phase 3 Soleno Therapeutics Not yet recruiting,
starts Spring 2018
Hyperphagia 8+ n/a ~100 15w 10+ US sites
Livoletide Phase 2b/3 Millendo Not yet recruiting,
starts Q4 2018
Hyperphagia 12-65 < 65 kg/m2  ~150 TBD  US & Europe, sites TBD
Carbetocin Phase 3 Levo Therapeutics Not yet recruiting,
starts Fall 2018
Hyperphagia, behavior  7-18  n/a  ~100 TBD 

US & other
sites TBD


Non-Medication and Previously Approved Drug Trials 

Study / Link Type Investigator Status Area of Research Ages BMI Length Site Locations

Mindfulness-based intervention for temper outbursts in PWS

Intervention Dr. Stewart Einfeld Recruiting Temper Outbursts 13-30 n/a 18 weeks

web based

Web-based study of cognition Exploratory   Dr. Carrie Bearden Recruiting Cognition and behavior 10-50 n/a 1.5 hours

web based

Does the gut microbiome differ between children with and without PWS?


Dr. Andrea Haqq Recruiting Gut microbiome 0-17 n/a 3 days North America (mail stool sample)
Parent-focused Remote Education to Enhance Development (PRETEND)

Social Intervention

Dr. Anastasia Dimitropoulos Recruiting Social behavior 3-9 n/a 8w

Remote with 2 in-person visits

Madison - April 21
Chicago -  April 22
Atlanta - mid-may

Developing Objective Biomarkers of Hyperphagia in Children with PWS


Dr. Anastasia Dimitropolous,
Dr. Alexandra Key
Recruiting Endpoint Development 3-12 n/a 1 visit

Case Western University, Vanderbilt University, 

Madison - April 21
Chicago -  April 22
Atlanta - mid-may

Oxytocin and the Autonomic Nervous System in PWS


Dr. Stewart Einfeld Recruiting Oxytocin 13-30 n/a 1 visit

University of Sydney,

Royal Children's Hospital, Victoria

Liraglutide Approved drug NovoNordisk Recruiting BMI - weight loss 6-18 not indicated 52w Baltimore, MD
Buffalo, NY International


Clinical Trials Overview Video


PWS Clinical Trials Directory

For more in-depth details about individual PWS clinical trials, visit our PWS Clinical Trials Directory.


Note that FPWR does not endorse or recommend any particular clinical study.  Please work with your doctor and the study sponsor to determine if a specific clinical study is right for you or your loved one with PWS.