Prader-Willi syndrome
MONDO:0008300Prader-Willi syndrome is a rare genetic disorder characterized by hypothalamic-pituitary abnormalities with severe hypotonia during the neonatal period and first two years of life and the onset of hyperphagia with a risk of morbid obesity during infancy and adulthood, learning difficulties and behavioral problems or severe psychiatric problems.
Also known as: Prader-Labhart-Willi syndrome, Prader-Willi syndrome, Prader-Willi-Labhart syndrome, Willi-Prader syndrome, PWS, Prader-Willi syndrome chromosome region, Prader-Willi-like syndrome associated with chromosome 6, obesity, muscular hypotonia, intellectual disability, short stature, hypogonadotropic hypogonadism, and small hands and feet
32 clinical trials for this condition and its sub-types, 31 tagged with Prader-Willi syndrome itself.
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Sub-types of Prader-Willi syndrome
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Schaaf-Yang syndrome 0 trials
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New drug under observation for rare genetic disorder
Disease control By invitation onlyThis 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.
Sponsor: Soleno Therapeutics, Inc. • Aim: Disease control
Last updated Aug 23, 2026 00:00 UTC
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Can early parent coaching help infants with rare genetic disorders thrive?
Disease control By invitation onlyThis 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…
Sponsor: RTI International • Aim: Disease control
Last updated Jul 15, 2026 00:00 UTC
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New drug pitolisant tested for safety in rare genetic disorder
Disease control By invitation onlyThis 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…
Phase 3 • Sponsor: Harmony Biosciences Management, Inc. • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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New drug aims to curb hunger in Prader-Willi syndrome
Disease control OngoingThis 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.
Phase 2 • Sponsor: Rhythm Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:04 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis 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…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
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Could a gentle nerve zap curb explosive outbursts in Prader-Willi syndrome?
Symptom relief OngoingThis 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 …
Phase 3 • Sponsor: Foundation for Prader-Willi Research • Aim: Symptom relief
Last updated Jun 27, 2026 12:04 UTC
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New nasal spray aims to tame relentless hunger in rare genetic disorder
Symptom relief OngoingThis 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 …
Phase 3 • Sponsor: ACADIA Pharmaceuticals Inc. • Aim: Symptom relief
Last updated Jun 26, 2026 16:16 UTC