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
31 clinical trials for this condition and its sub-types.
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Broader categories
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Growth hormone trial aims to boost muscle in Prader-Willi patients
Disease control CompletedThis completed Phase 3 study tested somatropin, a synthetic growth hormone, in 33 Japanese children and adults with Prader-Willi syndrome. The goal was to see if it safely improves body composition by increasing lean body mass and reducing fat. Participants were divided into thre…
Phase: PHASE3 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 08:11 UTC
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New lifestyle program aims to help Prader-Willi patients keep weight off
Disease control CompletedThis completed trial tested a personalized program of physical activity, nutrition guidance, and education for 128 adults with Prader-Willi syndrome. Participants followed the program during a 5-week hospital stay, with the goal of stabilizing or reducing their body mass index (B…
Phase: NA • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC
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Can a wakefulness drug beat daytime sleepiness in Prader-Willi syndrome?
Symptom relief CompletedThis phase 2 study tested whether pitolisant, a drug that promotes wakefulness, can safely reduce excessive daytime sleepiness in people with Prader-Willi syndrome. The trial enrolled 65 participants aged 6 to 65 and compared pitolisant to a placebo over 11 weeks, followed by an …
Phase: PHASE2 • Sponsor: Harmony Biosciences Management, Inc. • Aim: Symptom relief
Last updated Jun 27, 2026 12:31 UTC
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New parent support programme shows promise for families of children with complex needs
Symptom relief CompletedThis pilot study tested a community-based group programme called 'Encompass' for parents of children under 5 with complex neurodisability. Fifteen parents in East London attended ten group sessions over six months. The study aimed to see if the programme was feasible and acceptab…
Phase: NA • Sponsor: City, University of London • Aim: Symptom relief
Last updated Jun 27, 2026 12:05 UTC
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Can a 'Love Hormone' spray tame Prader-Willi symptoms?
Symptom relief CompletedThis study tested whether oxytocin nasal spray, sometimes called the 'love hormone,' can improve behavior and eating problems in adults with Prader-Willi syndrome. Fifty participants received either oxytocin or a placebo daily or every three days for 28 days. Researchers measured…
Phase: PHASE2, PHASE3 • Sponsor: University Hospital, Toulouse • Aim: Symptom relief
Last updated Jun 27, 2026 08:02 UTC
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Rare disease mystery: NIH launches deep dive into Smith-Magenis syndrome
Knowledge-focused CompletedThis study follows nearly 600 people with Smith-Magenis syndrome (SMS), a rare genetic condition, to track how their health, behavior, and development change over time. Researchers will perform detailed medical exams, genetic tests, and surveys to better understand the syndrome's…
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Aug 15, 2026 00:00 UTC
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Hormone study aims to unlock Prader-Willi feeding mystery
Knowledge-focused CompletedThis completed study looked at how appetite-regulating hormones change in infants with Prader-Willi syndrome during the first four years of life. Researchers collected blood samples from 215 infants to understand why they switch from poor feeding to severe overeating and obesity.…
Phase: NA • Sponsor: University Hospital, Toulouse • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:05 UTC