Can a wakefulness drug beat daytime sleepiness in Prader-Willi syndrome?

NCT ID NCT04257929

Completed Symptom relief Sponsor: Harmony Biosciences Management, Inc. Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Prader-Willi syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PRADER-WILLI SYNDROME

Contacts and locations

Locations

Ann and Robert H Lurie Children's Hospital

Chicago, Illinois, 60611, United States
CTI

Cincinnati, Ohio, 45212, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Johns Hopkins School of Medicine

Baltimore, Maryland, 21205, United States
Nemours Alfred I duPont Hospital for Children

Wilmington, Delaware, 19803, United States
Rady Children's Hospital - San Diego

San Diego, California, 92123, United States
Road Runner Research

San Antonio, Texas, 78249, United States
Santa Monica Clinical Trials

Santa Monica, California, 90404, United States
Sleep Medicine Specialists of California

San Ramon, California, 94583, United States
Texas Children's Hospital/Baylor College of Medicine

Houston, Texas, 77030, United States
University of Florida College of Medicine

Gainesville, Florida, 32608, United States
University of Nebraska Medical Center

Omaha, Nebraska, 68114, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37203, United States

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