Non-acquired pituitary hormone deficiency

This study follows about 200 people with Prader-Willi syndrome who are taking or starting VYKAT XR. Researchers will track side effects and how the drug affects their health over time. The goal is to gather more safety information, not to test if the drug cures the condition.

This study tests a program called PIXI that coaches parents of infants diagnosed with rare neurogenetic disorders (such as Fragile X, Angelman, or Down syndrome) during the first year of life. The program combines education about the disorder, guided parent-child interaction acti…

This study follows 200 children in Japan with growth hormone deficiency who are taking Sogroya® (somapacitan) as part of their normal care. Researchers will monitor side effects and growth over 1 to 3 years to see how safe and effective the drug is in real-world use. Participants…

This phase 3 trial is testing the safety of pitolisant in 150 people with Prader-Willi syndrome who have already taken the drug in a previous study. Participants will receive pitolisant tablets and be monitored for side effects. The goal is to see if pitolisant is safe for long-t…

This phase 2 trial tests setmelanotide (Imcivree), a daily injection, in 18 people aged 6 to 65 with Prader-Willi syndrome who have obesity. The study will check if the drug is safe and helps reduce body weight and hunger over 52 weeks.

This study tracks the safety and effectiveness of Ngenla, a growth hormone injection, in over 500 children aged 3 and older with growth hormone deficiency in Korea. Researchers will monitor side effects and growth changes over 6 years during routine medical care. The treatment he…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This phase 3 study tests a device that gently stimulates the vagus nerve through the skin (tVNS) to see if it can safely reduce temper outbursts in people with Prader-Willi syndrome. About 102 participants aged 10 to 40 will use either continuous or intermittent stimulation. The …

This study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …

This Phase 3 trial tests a nasal spray called carbetocin to reduce the intense, constant hunger (hyperphagia) in people with Prader-Willi syndrome. The study involves 170 participants aged 5 to 30 and lasts 12 weeks. Caregivers will rate changes in hunger-related behaviors using …

This 10-year observational study will follow 500 children with growth hormone deficiency who are receiving the once-weekly injection somapacitan as part of their routine care. Researchers will track side effects, medication errors, and new cases of cancer or type 2 diabetes. The …

This study looks at children and teenagers with growth hormone deficiency or who were born small for their age. It tracks how closely they follow their prescribed Norditropin® treatment and whether that affects their final height. The study is observational, meaning no new treatm…