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!
|Drug / link||Phase||Sponsor||Status||Primary Target||Ages||BMI / WT||# Patients||Length||Site Locations|
|Phase 3||Levo Therapeutics||Top-Line Results Available||Hyperphagia, behavior||7-18||n/a||~175||8w dbl blind + 56w extension||n/a|
|Phase 2||Harmony Biosciences||Not Yet Recruiting 2020||Excessive Daytime Sleepiness||6 - 65||n/a||60||11 dbl blind + optional open label extension||TBA|
|Phase 3||Soleno Therapeutics||Top-Line Results Available||Hyperphagia||4+||20* - 130 kg||~100||15w dbl blind + 9m extension||n/a|
|Phase 4||NYU Langone Health||Recruiting||Aggression, self-injurious behavior||6 - 35||n/a||33||19 w||
|Phase 2||Montefiore Medical Center||Coming Soon||Behavior||5 - 30||n/a||26||12 w||
|Oxytocin||Phase 2||Montefiore Medical Center||Recruiting||Hyperphagia, Repetitive Behavior||5 - 17||n/a||50||8w||NY|
|Tesomet||Phase 2, Part 2||Saniona||complete||Hyperphagia, Weight loss||12-17||BMI > 85th percentile||10-15||12w||n/a|
Click on the pins for information about trials that are currently recruiting.
|Study / Link||Type||Investigator||Status||Area of Research||Ages||Length||Site Locations|
|Interview||Covance||Recruiting||Daytime Sleepiness||6 - 65||1 90-minute interview||virtual|
|Intervention||Dr. Forster||Recruiting||Family Stress||13 - 18||4 sessions||web based|
|Exploratory||Dr. Jill Hamilton||Recruiting||Stress and Appetite||7 - 16||2 visits||SickKids
|Natural History||Theresa Strong, PhD||Ongoing - not recruiting||Serious Medical Events||5+||4 years||virtual|
|Intervention||Dr. Stewart Einfeld||Recruiting||Temper Outbursts||13-30||18 weeks||
|Parent-focused Remote Education to Enhance Development (PRETEND)||
|Dr. Anastasia Dimitropoulos||Recruiting||Social behavior||6-9||8w||
Remote with 2 in-person visits
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.