New hope for wakefulness: drug fights sleepiness in rare genetic disorder

NCT ID NCT04257929

First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 31 times

Summary

This study tested a medication called pitolisant to see if it could safely reduce severe daytime sleepiness in people with Prader-Willi syndrome, a genetic condition. 65 participants aged 6 to 65 took either the drug or a placebo for 11 weeks. The main goal was to measure changes in sleepiness using a standard questionnaire. The trial is now complete.

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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

Conditions

Explore the condition pages connected to this study.