Can a wakefulness drug beat daytime sleepiness in Prader-Willi syndrome?
NCT ID NCT04257929
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This 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 open-label extension. The main goal was to see if pitolisant improves sleepiness scores based on caregiver reports.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
pitolisant (oral tablet)
What this could lead to
If it works, this could provide a new option to help people with Prader-Willi syndrome stay awake and alert during the day.
What could go wrong
This is an early-phase study with only 65 participants, so results may not apply to everyone. The drug may not work better than a placebo, and side effects are possible.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Ann and Robert H Lurie Children's Hospital
Chicago, Illinois, 60611, United States
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CTI
Cincinnati, Ohio, 45212, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Johns Hopkins School of Medicine
Baltimore, Maryland, 21205, United States
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Nemours Alfred I duPont Hospital for Children
Wilmington, Delaware, 19803, United States
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Rady Children's Hospital - San Diego
San Diego, California, 92123, United States
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Road Runner Research
San Antonio, Texas, 78249, United States
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Santa Monica Clinical Trials
Santa Monica, California, 90404, United States
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Sleep Medicine Specialists of California
San Ramon, California, 94583, United States
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Texas Children's Hospital/Baylor College of Medicine
Houston, Texas, 77030, United States
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University of Florida College of Medicine
Gainesville, Florida, 32608, United States
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University of Nebraska Medical Center
Omaha, Nebraska, 68114, United States
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Vanderbilt University Medical Center
Nashville, Tennessee, 37203, United States