PRADER-WILLI SYNDROME

This study tests a weekly injection called RM-718 in healthy people with obesity and in patients with obesity caused by problems in the MC4R pathway, including hypothalamic obesity and Prader-Willi syndrome. The main goal is to check the drug's safety and how the body processes i…

This early-stage trial tests a new drug called CSTI-500 in 12 people aged 13 to 50 with Prader-Willi syndrome, a genetic condition that causes severe overeating and behavioral issues. The study aims to see if the drug is safe and can reduce excessive hunger, with doses adjusted b…

This study tests a weight-loss medication called tirzepatide in young adults aged 18-26 with Prader-Willi syndrome, hypothalamic obesity, or general obesity. Participants will take the drug for one year to see how much weight they lose and how their health changes. The goal is to…

This study tests whether probiotics and prebiotics can safely help control appetite and weight gain in 60 children with Prader-Willi syndrome, a rare genetic condition that causes severe obesity. Researchers will measure changes in body mass index, body fat, and gut bacteria over…

This study tests a group program to help teens with Prader-Willi Syndrome (PWS) and their families handle emotional challenges like irritability, meltdowns, and anxiety. Ten teens aged 13-17.5 with a confirmed PWS diagnosis will attend weekly 90-minute sessions. The goal is to im…

This study tests a drug called pitolisant to see if it can reduce severe daytime sleepiness in people with Prader-Willi syndrome, a genetic condition. About 134 participants aged 6 and older will receive either the drug or a placebo, and researchers will also check if it helps wi…

This study tests whether bright light therapy can help reduce excessive daytime sleepiness in people with Prader-Willi syndrome. About 50 participants will receive either bright light or a placebo light to see if it improves alertness, mood, and behavior. The goal is to find a si…

This study will follow 2000 children and adults with rare genetic conditions called imprinting disorders, such as Silver-Russell and Prader-Willi syndromes. Researchers want to find common metabolic patterns and health risks like obesity or diabetes. No new treatments are tested;…

This study tests a program called PACE that helps adults with intellectual disability become more physically active. About 120 adults, along with their caregivers and coaches, will take part. The program focuses on increasing daily steps and moderate-to-vigorous activity, and res…

This study tests a noninvasive brain stimulation technique called TMS to learn more about hunger and fullness in people with Prader-Willi syndrome. About 20 participants will receive TMS and have brain scans to see how their brain activity changes. The goal is to understand if TM…

This study is creating a detailed registry of people with rare diseases like amyloidosis, sarcoidosis, and many others. Researchers will collect information from medical records and patient reports to learn how these diseases progress, how they are treated, and what outcomes pati…