This guest blog was contributed by Sara Cotter, CEO, Levo Therapeutics.
When developing new medicines to treat PWS, companies need to demonstrate meaningful improvements in PWS symptoms. For growth hormone, this was relatively straightforward because changes in height are easily measured. For behavioral or psychiatric symptoms in PWS, this becomes a far more challenging task.
As a community, we are so fortunate to have the Hyperphagia Questionnaire for Clinical Trials, also known as the HQ-CT. This hyperphagia questionnaire was originally developed by Elisabeth Dykens and colleagues at Vanderbilt University, and further adapted for clinical trials by Zafgen and RTI Health Solutions. It measures observable behaviors related to hyperphagia, with questions like: “How upset did the person generally become when denied a desired food?” and “How often did the person try to sneak or steal food (that you are aware of)?”That said, the HQ-CT is only intended to measure a portion of PWS symptoms. For example, many of our children suffer from significant anxiety – both food- and non-food related – which is not measured by the HQ-CT. In fact, clinicians and researchers have struggled to find any questionnaire that adequately measures anxiety in PWS, given its unique features. Without this type of instrument, we can’t assess whether a new medicine helps treat anxiety in PWS.
Levo Therapeutics is working to change this by developing a new tool called the PWS Anxiety and Distress Questionnaire (PADQ). This anxiety questionnaire measures observable behaviors related to anxiety, and it reflects the collective experience of PWS parents and caregivers. The PADQ was created with support from FPWR and RTI Health Solutions.
Now we’re hoping you can help, too, by completing a 5–10 minute questionnaire on the PWS Global Registry.
If you're the primary caregiver for a person with PWS, age 5 or older, by completing this questionnaire you can help develop the PADQ and measure anxiety directly for the first time in our community.
- If you're enrolled in the Global PWS Registry, please log in here, click Take Surveys next to the PWS participant’s name, and complete the Anxiety and Distress survey.
- If you're not already enrolled in the PWS Registry, go to the the registry web page and click the Register button. Set up your account, complete the Getting Started survey, and then complete the Anxiety and Distress survey.
You'll be asked to complete the questionnaire so twice; once to start and then a second time two weeks later.
Together we can shed light on this important aspect of PWS behavior!
Learn more about PWS clinical trials by visiting our updated PWS Clinical Trials page.