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 BMI / WT # Patients Length Site Locations

Pitolisant

Phase 2 Harmony Biosciences Now Enrolling Excessive Daytime Sleepiness 6 - 65 n/a 60 11 dbl blind + optional open label extension CA, CO, DE, IL, FL, MD, TN, TX, UT,

Guanfacine

Phase 4 FPWR Enrolling January 2021 Aggression, self-injurious behavior 6 - 35 n/a 33 19 w

NY

Cannabidivarin (CBDV)

Phase 2 Montefiore Medical Center Now Enrolling Behavior 5 - 30 n/a 26 12 w

NY

Carbetocin

Phase 3 Levo Therapeutics Complete Top-Line Results Available  Hyperphagia, behavior    7-18  n/a complete complete complete

DCCR

Phase 3 Soleno Therapeutics  Complete Top-Line Results Available  Hyperphagia  4+ 20* - 130 kg complete complete complete
Tesomet Phase 2, Part 2 Saniona

Phase 2 complete

2b Coming Soon

 Hyperphagia, Weight loss  12-17   BMI > 85th percentile   10-15 12w   n/a

Find a Trial Near You!

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

Non-Medication PWS Studies

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

Chronic Stress, Cognition and Food Cue Reactivity

Exploratory Dr. Jill Hamilton Recruiting Stress and Appetite 7 - 16 2 visits SickKids
Toronto, CA

Exercise for Young People with PWS

Intervention Prof Nora Shields Recruiting Exercise 13+ 24 weeks Sydney, Australia

PATH for PWS

Natural History Theresa Strong, PhD Ongoing - not recruiting Serious Medical Events 5+ 4 years virtual

Mindfulness-based intervention for temper outbursts in PWS

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

web based

Parent-focused Remote Education to Enhance Development (PRETEND)

Social Intervention

Dr. Anastasia Dimitropoulos Recruiting Social behavior 6-9 8w

Remote with 2 in-person visits

 

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