Prader-Willi syndrome
Clinical trials for Prader-Willi syndrome explained in plain language.
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Can a pill tame the relentless hunger of Prader-Willi syndrome?
Disease control CompletedThis phase 2 trial tests an oral drug called ARD-101 in adults with Prader-Willi syndrome, a genetic condition that causes an unrelenting feeling of hunger. Researchers give the drug twice daily for 28 days to see if it safely reduces hyperphagia, the intense drive to eat, using …
Matched conditions: PRADER-WILLI SYNDROME
Phase 2 • Sponsor: Aardvark Therapeutics, Inc. • Aim: Disease control
Last updated Sep 10, 2026 00:00 UTC
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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…
Matched conditions: PRADER-WILLI SYNDROME
Phase 3 • 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…
Matched conditions: PRADER-WILLI SYNDROME
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 …
Matched conditions: PRADER-WILLI SYNDROME
Phase 2 • Sponsor: Harmony Biosciences Management, Inc. • Aim: Symptom relief
Last updated Jun 27, 2026 12:31 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…
Matched conditions: PRADER-WILLI SYNDROME
Phase 2/3 • 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…
Matched conditions: PRADER-WILLI SYNDROME
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Sep 11, 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.…
Matched conditions: PRADER-WILLI SYNDROME
Sponsor: University Hospital, Toulouse • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:05 UTC