Study Purpose If you are the caregiver and the parent or legal guardian of someone aged 6 to 65 years of age who has been diagnosed with Prader-Willi syndrome (PWS) and who also experiences daytime sleepiness, you and your care recipient may qualify for a 90-minute video interview that will pay you $100 for your participation.
Understanding the Pathophysiology of Schaaf-Yang Syndrome to Improve Care and Treatment Options Dr. Fleischer at Southern Illinois University School of Medicine, in conjunction with Dr. Schaaf from Heidelberg University in Germany, is conducting a study entitled, “Understanding the Pathophysiology of Schaaf-Yang Syndrome to Improve Care
Study Purpose The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Prader-Willi syndrome (PWS) ages 6 to 65 years. Secondary study objectives are to evaluate the impact of pitolisant on: Behavioral symptoms Cognitive function Safety and effectiveness of long-term treatment Caregiver burden The study will consist of a Screening Period, an 11-week Double-Blind Treatment Phase (including a 3-week Titration Period and an 8-week Stable Dose Period), and an optional Open Label Extension (OLE) Phase. The OLE Phase will be multi-year in duration and will continue until either pitolisant is approved for patients with PWS or the Sponsor elects to terminate the study.
We want to learn more about how stress affects appetite in Prader-Willi Syndrome and how different areas of the brain communicate with one another in adolescents. With your help, we can gain more insight into the functioning of the adolescent brain.
Study Purpose The Acceptance and Commitment Training (ACT) Study will examine the effectiveness of ACT training for fathers of adolescents ages 13-18 with PWS with the goal of improving life satisfaction, reducing stress and improving family functioning. This remote study will use Zoom video conferencing to provide 4 sessions of ACT training. The study is seeking fathers who have an adolescent with PWS age 13-18 to participate in a 4-session, on-line, small group training exercise to reduce father’s perception of stress, improve parenting satisfaction and increase family involvement. Participants must reside at home with their adolescent and the adolescent’s mother, have a personal computer with a camera, microphone and internet access, and agree to privacy and confidentiality of shared information. They must be able to read and speak English. Sessions will be scheduled to accommodate 8 fathers per group. After an initial 90 minute session, three additional 60 minute training sessions will occur at a frequency of every other week. Additional “on-your-own” time will be needed to practice newly acquired skills. Both fathers and mothers will be required to participate in a brief screening interview by phone and then complete several on line questionnaires at the outset, during the study, and at the conclusion of the study. These questionnaires will assess stress, family function and behaviors unique to the adolescent with PWS.
Study Purpose This phase 2, randomized, double blind 12-week treatment trial of Cannabidivarin (CBDV) will assess the affect of CBDV on behavior in people with PWS.
Got Anxiety? Want to help pave the way for new medicines to treat anxiety in PWS? Levo Therapeutics and the Foundation for Prader-Willi Research are working to develop a new questionnaire for use in clinical studies of novel treatments for PWS. We want to know more about how anxiety is expressed in people with PWS.
Study Purpose Soleno Therapeutics is conducting a Phase 3 study of DCCR to measure the efficacy of the DCCR for treating hyperphagia in PWS. The study seeks to enroll 105 patients with PWS ages 4 years and older. The study will be a 15 week, randomized, double-blind, placebo-controlled study of Diazoxide Choline Controlled-Release Tablet (DCCR) and will require a once-daily oral administration. The initial 15-week study will be followed by a 9-month extension during which time all patients will receive DCCR. Protocol Update: A recent amendment to the study protocol now allows patients between 20kg and 130kg to enroll in the study, provided they meet all eligibility criteria. Individual study sites will be able to enroll patients in the new weight band (20-30kg) once the appropriate tablet strengths come available, which will likely be in the September timeframe. Patients who are interested in enrolling should contact their nearest study coordinator.
[NOTE: This study is now closed. Learn about the results here.] FPWR is conducting a text-messaging based research study to assess weight changes in Prader-Willi syndrome (PWS) over a period of six months. We’d like to learn more about the normal variation in weight, over time, in the PWS population. We hope to recruit 150-300 individuals across the US and Canada to participate, so we can get a good picture of how weight changes over time. We anticipate that this study will inform future clinical trials for hyperphagia/obesity related therapies, and provide a basis for understanding how well potential therapies are working. Patients should not be enrolled in other clinical trials for the duration of this study. The study can be done from your home, with no visit to a clinic.
Study Purpose This phase 2 randomized double blind 8-week treatment trial of intranasal oxytocin (IN-OXT) will assess IN-OXT's affect on: eating behaviors, repetitive behaviors, weight and body composition, quality of life, and salivary OXT and hormone levels. Data from the study will add to the current knowledge that OXT is an effective treatment for hyperphagia as well as other symptoms of PWS.
