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


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 Recruiting  Hyperphagia 8 - 17 n/a 66 12w

Tacoma, WA
San Diego, CA*
Iowa City, IA*
Baltimore, MD*
Tuscon, AZ*
Boston, MA*
Memphis, TN*

*coming soon


Phase 2 Montefiore Medical Center Recruiting Hyperphagia, Repetitive Behavior 5 - 17 n/a 50 8w Bronx, NY


Phase 3 Soleno Therapeutics Recruiting Hyperphagia 8+ n/a ~100 15w

Palo Alto, CA
San Diego, CA
Seattle, WA
Saint Paul, MN
Gainesville, FL
More TBA

Tesomet Phase 2, Part 2  Saniona Recruiting   Hyperphagia, Weight loss  12-17   BMI > 85th percentile   10-15 12w   Prague, Czechia
Liraglutide Approved NovoNordisk Recruiting BMI - weight loss 6-18 30 kg/m2 or BMI > 95th percentile   52w Baltimore, MD
Buffalo, NY International
Livoletide Phase 2b/3 Millendo coming soon Hyperphagia 12-65 < 65 kg/m2  ~150 TBD 

US & Europe, sites TBA

Phase 3 Levo Therapeutics coming soon Hyperphagia, behavior  7-18  n/a  ~150 TBD 

US & Canada
sites TBA

Find a Trial Near You!

Click on the pins for more trial information.

Non-Medication PWS Studies

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

A Study to Assess Weight Change Over 6 months in Prader-Willi Syndrome

Natural History Dr. Theresa Strong Recruiting Weight Change 12 and up n/a 6 months Virtual across North America

PWS Anxiety and Stress Questionnaire (PADQ)

Survey Levo & FPWR Recruiting Anxiety & Stress 5 and up n/a 10 minutes Virtual

PWS Activity, Sleep and Eating Pattern Pilot Study

Exploratory Dr. Panda Recruiting Activity, Sleep and Eating 13 - 65 n/a 2 weeks Virtual

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


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, Vanderbilt

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

PWS Clinical Trials Overview Video


How Patients Can Partner

Download PWS Clinical Trial Webinar Slides

PWS Clinical Trials Directory

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


FPWR does not endorse or recommend any specific study. Our goal is to provide information to help 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. 

PWS Clinical Trials FAQ

Download the PWS Clinical Trials FAQ Sheet