PWS Clinical Trials

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 learn more about the drug development process on our blog: Stages of PWS Drug Development Overview. 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!

Harper_and_Daddy

Sign Up For PWS Clinical Trial Alerts

PWS Clinical Trials In Progress / Starting Soon

Drug / link Phase Sponsor Status Primary Target Ages Length Site Locations

VNS4PWS

Phase 3 FPWR Enrolling Behavior Outbursts 10-40 12 mo

CA, GA, NY, MO, TX 
others coming soon

Pitolisant

Phase 3 Harmony Biosciences Enrolling Excessive Daytime Sleepiness 6 - 65  

CA, CO, DE, GA, OH, NY, TX
Others coming soon

Carbetocin

Phase 3 Acadia Enrolling Hyperphagia 5-30 19 w

AL, AZ, CA, CO, IA, IL, KY, MA, MO, NY, OH, PA, TN, TX, UT, WA, CANADA

Light Therapy

Phase 4 FPWR Enrolling Daytime Sleepiness 6 + 3 w

REMOTE

TMS

Interventional FPWR Enrolling Hyperphagia 18-64 3 w

MA

Cannabidivarin (CBDV)

Phase 2 Montefiore Medical Center Enrolling Behavior 5 - 30 12 w

NY

ARD-101

Phase 2 Aardvark Therapeutics complete Hyperphagia 17-65 4 w CA, CO

RGH-706

Phase 2 Gedeon Richter Enrollment
Complete
Hyperphagia 17+  

Enrollment
Complete

DCCR

Phase 3 Soleno Therapeutics  Complete Top-Line Results Available  Hyperphagia  4+ complete complete

Find a Trial Near You!

Click on the pins for information about trials that are currently recruiting.

Non-Medication PWS Studies

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

PWS | SYS Biorepository

FPWR & CombinedBrain Recruiting Urine and Blood Samples All 1 visit Philadelphia, Seattle, Atlanta, Kansas City, Los Angeles

Skin Picking Study

Dr. Scott Hall Recruiting Skin Picking 12-17 1 visit Stanford, CA

WellCast: Supporting Wellbeing of Caregivers Via Telehealth

Dr. Bridgette Kelleher Recruiting Support for caregivers 2-35 varies remote

PRETEND Study Dissemination

Dr. Anastasia Dimitropoulos Recruiting Intervention Curriculum 8-20 n/a remote

Global PWS Registry

FPWR Enrolling Natural History ALL ongoing virtual

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

Want to learn more about clinical trials? Download our PWS Clinical Trials FAQ Sheet.