New drug aims to tackle sleepiness in rare genetic disorder

NCT ID NCT06366464

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

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 3 trial tests whether pitolisant can reduce excessive daytime sleepiness in people aged 6 and older with Prader-Willi syndrome. About 134 participants will receive either pitolisant or a placebo, and researchers will also look at effects on behavior and appetite. The study is currently recruiting.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: pitolisant
What this could lead to: If successful, pitolisant could become a new treatment to reduce daytime sleepiness and improve behavior in people with Prader-Willi syndrome.
What could go wrong: This is a Phase 3 trial, but results are not guaranteed. The drug may not work better than 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.

Disorders of Excessive Somnolence Prader-Willi syndrome Problem Behavior

As listed by the trial registrant

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

PRADER-WILLI SYNDROME

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••
Contact

Email: •••••@•••••

Locations

AMNDX Inc.
RECRUITING

Thornhill, Ontario, Canada
Aarhus University Hospital
RECRUITING

Aarhus, Denmark
Ann And Robert H Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Atlanta Diabetes Associates
WITHDRAWN

Atlanta, Georgia, 30318, United States
Azienda Ospedale Università Padova - Dipartimento Salute della Donna e del Bambino - INCIPIT - PIN
RECRUITING

Padova, Italy
Azienda Ospedaliero Universitaria A Meyer
RECRUITING

Florence, Italy
CHU d'Angers
RECRUITING

Angers, France
CHU de Toulouse-Hôpital Des Enfants
RECRUITING

Toulouse, France
CTI Clinical Research Center
WITHDRAWN

Cincinnati, Ohio, 45212, United States
Center for Human Genetics
RECRUITING

Cleveland, Ohio, 441016, United States
Center of Excellence in Diabetes and Endocrinology
RECRUITING

Sacramento, California, 95821-2123, United States
Children's Hospital at Westmead
RECRUITING

Westmead, Australia
Childrens Hospital of Wisconsin
RECRUITING

Milwaukee, Wisconsin, 53226, United States
Colorado Children's Hospital
RECRUITING

Aurora, Colorado, 80045, United States
Corporacio Sanitaria Parc Tauli, Sabadell
RECRUITING

Barcelona, Spain
Emory University School of Medicine
RECRUITING

Atlanta, Georgia, 30322, United States
Fulbourn Hospital
RECRUITING

Cambridge, England, United Kingdom
Hospital Sant Joan de Deu
RECRUITING

Barcelona, Spain
Hospital Universitario 12 de Octubre
RECRUITING

Madrid, Spain
Hull Royal Infirmary
RECRUITING

Hull, England, United Kingdom
IRCCS Materno Infantile Burlo Garofolo - INCIPIT - PIN
RECRUITING

Trieste, Italy
Institutul National de Endocrinologie C. I. Parhon
RECRUITING

Bucharest, Romania
Institutul National de Endocrinologie C. I. Parhon
RECRUITING

Bucharest, Romania
Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN
RECRUITING

Genova, Italy
Jodha Tishon Inc.
RECRUITING

Toronto, Ontario, Canada
Johns Hopkins Hospital
RECRUITING

Baltimore, Maryland, 21224, United States
Karolinska Universitetssjukhuset Solna
RECRUITING

Solna, Sweden
Louis Turcanu Emergency Clinical Hospital for Children
RECRUITING

Timișoara, Romania
Maimonides Medical Center
RECRUITING

Brooklyn, New York, 11219, United States
Mayo Clinic-PPDS
RECRUITING

Rochester, Minnesota, 55905, United States
Montefiore Medical Center
RECRUITING

The Bronx, New York, 10467, United States
National Clinical Center for Children Neurorehabilitation "Dr. Nicolae Robanescu"
RECRUITING

Bucharest, Romania
Nemours Children's Hospital
RECRUITING

Wilmington, Delaware, 19803-3607, United States
Ninewells Hospital - PPDS
RECRUITING

Dundee, Scotland, United Kingdom
Ospedale Pediatrico Bambino Gesù IRCCS
RECRUITING

Roma, Italy
Ospedale San Raffaele S.r.l. - PPDS
RECRUITING

Milan, Italy
Queensland Children's Hospital
RECRUITING

Brisbane, Queensland, Australia
Rady Children's Hospital - Scan Diego
RECRUITING

San Diego, California, 92123, United States
Rare Disease Research
WITHDRAWN

Atlanta, Georgia, 30329, United States
Rigshospitalet
RECRUITING

Copenhagen, Denmark
Riley Children's Hospital
RECRUITING

Indianapolis, Indiana, 46202, United States
Road Runner Research
RECRUITING

San Antonio, Texas, 78249-3539, United States
Royal Hospital for Children and Young People
RECRUITING

Edinburgh, Scotland, United Kingdom
Royal Prince Alfred Hospital
RECRUITING

Camperdown, Australia
Samodzielny Publiczny Szpital Kliniczny
RECRUITING

Szczecin, Poland
Santa Monica Clinical Trials
RECRUITING

Los Angeles, California, 90025, United States
Science 37 (at-home option)
RECRUITING

Morrisville, North Carolina, 27560, United States
Sydney Children's Hospital
RECRUITING

Randwick, Australia
Texas Children's Hospital
RECRUITING

Houston, Texas, 77030, United States
Texas Valley Clinical Research
TERMINATED

Weslaco, Texas, 78596, United States
UZ Brussels
RECRUITING

Jette, Belgium
Universitario Virgen de la Victoria
RECRUITING

Málaga, Spain
University Hospital Essen
RECRUITING

Essen, Germany
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu-Chalubinskiego 2-2a
RECRUITING

Wroclaw, Poland

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