Clinical trials are carefully designed research studies. They are a critical step in determining whether a treatment works and is safe for people. Each study follows strict guidelines and is reviewed by ethics committees (IRBs) to protect participants. Participation is always voluntary.
In recent years, families in our community have played a key role in advancing promising treatments — such as VYKAT XR— through clinical trial participation. Every trial — even those that don’t lead to approval — helps us learn and get closer to better treatments.
Click the logos to learn more about each trial.
The Global PWS Registry is one of the most important tools we have for driving research forward. It helps researchers and clinicians better understand how PWS affects individuals at different ages, and it provides valuable data to support clinical trials and the approval of new treatments.
In 2025, Vykat XR became the first FDA-approved treatment for hyperphagia in individuals with PWS, and data from the Global PWS Registry played an important role in this historic approval. Families who participated in the Registry completed surveys about hunger-related behaviors. This real-world evidence showed that individuals with PWS who were not taking DCCR had higher hyperphagia scores than those who participated in the Phase 3 DCCR trial — reinforcing the drug’s effectiveness.
Continued Registry participation will support future clinical trials and approval efforts for other promising treatments. How does the registry support trials?
Be part of the next breakthrough! Join the Global PWS Registry and help power future discoveries, improve care, and advance safer and effective treatments for people with PWS.
The Foundation for Prader-Willi Research (federal tax id 31-1763110) is a nonprofit corporation with federal tax exempt status as a public charity under section 501(c)(3).
