RECRUITING Phase 3 ClinicalTrials.gov

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Sponsor: Harmony Biosciences Management, Inc. • ClinicalTrials.gov ID: NCT06366464

Registry updated: 2026-08-25

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Description

This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome. The primary objective of this study is to evaluate the efficacy of pitolisant in treating excessive daytime sleepiness (EDS) in patients ≥6 years of age with Prader-Willi syndrome. Secondary objectives include assessing the impact of pitolisant on: Irritable and disruptive behaviors Hyperphagia Other behavioral problems including social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech

Interventions

Pitolisant tablet (Drug)
Pitolisant tablet
Placebo tablet (Other)
Placebo tablet

Eligibility criteria

Inclusion Criteria: * Genetically confirmed diagnosis of PWS * Excessive daytime sleepiness * Has a consistent parent/caregiver (preferably the same person throughout the study) who is willing and able to complete the required study assessments. * In the opinion of the Investigator, the patient/parent(s)/caregiver(s)/legal guardian(s) are capable of understanding and complying with the requirements of the protocol and administration of oral study drug. Exclusion Criteria: * Has a diagnosis of sleep apnea (OSA, CSA) that is not adequately controlled * Has a diagnosis of hypersomnia due to another sleep/medical disorder * Participation in an interventional research study involving another investigational medication, device, or behavioral treatment within 30 days or 5 half-lives (whichever is longer) of the investigational medication prior to Screening

Conditions

Prader-Willi Syndrome

Locations

Santa Monica Clinical Trials — Los Angeles, California, United States
Center of Excellence in Diabetes and Endocrinology — Sacramento, California, United States
Rady Children's Hospital - Scan Diego — San Diego, California, United States
Colorado Children's Hospital — Aurora, Colorado, United States
Nemours Children's Hospital — Wilmington, Delaware, United States
Atlanta Diabetes Associates — Atlanta, Georgia, United States
Emory University School of Medicine — Atlanta, Georgia, United States
Rare Disease Research — Atlanta, Georgia, United States
Ann And Robert H Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
Riley Children's Hospital — Indianapolis, Indiana, United States
Johns Hopkins University Hospital — Baltimore, Maryland, United States
Johns Hopkins Hospital — Baltimore, Maryland, United States
Mayo Clinic-PPDS — Rochester, Minnesota, United States
Maimonides Medical Center — Brooklyn, New York, United States
Montefiore Medical Center — The Bronx, New York, United States
Science 37 (at-home option) — Morrisville, North Carolina, United States
Science 37 — Morrisville, North Carolina, United States
CTI Clinical Research Center — Cincinnati, Ohio, United States
Center for Human Genetics — Cleveland, Ohio, United States
Texas Children's Hospital — Houston, Texas, United States
Road Runner Research — San Antonio, Texas, United States
Texas Valley Clinical Research — Weslaco, Texas, United States
Childrens Hospital of Wisconsin — Milwaukee, Wisconsin, United States
Queensland Children's Hospital — Brisbane, Queensland, Australia
Royal Prince Alfred Hospital — Camperdown, Australia
Sydney Children's Hospital — Randwick, Australia
Children's Hospital at Westmead — Westmead, Australia
UZ Brussels — Jette, Belgium
AMNDX Inc. — Thornhill, Ontario, Canada
Jodha Tishon Inc. — Toronto, Ontario, Canada
Aarhus University Hospital — Aarhus, Denmark
Rigshospitalet — Copenhagen, Denmark
CHU d'Angers — Angers, France
CHU de Toulouse-Hôpital Des Enfants — Toulouse, France
University Hospital Essen — Essen, Germany
University of Florence — Florence, Tuscany, Italy
Azienda Ospedaliero Universitaria A Meyer — Florence, Italy
Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN — Genova, Italy
Ospedale San Raffaele S.r.l. - PPDS — Milan, Italy
Azienda Ospedale Università Padova - Dipartimento Salute della Donna e del Bambino - INCIPIT - PIN — Padova, Italy
Ospedale Pediatrico Bambino Gesù IRCCS — Roma, Italy
IRCCS Materno Infantile Burlo Garofolo - INCIPIT - PIN — Trieste, Italy
Samodzielny Publiczny Szpital Kliniczny — Szczecin, Poland
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu-Chalubinskiego 2-2a — Wroclaw, Poland
Institutul National de Endocrinologie C. I. Parhon — Bucharest, Romania
Institutul National de Endocrinologie C. I. Parhon — Bucharest, Romania
National Clinical Center for Children Neurorehabilitation "Dr. Nicolae Robanescu" — Bucharest, Romania
Louis Turcanu Emergency Clinical Hospital for Children — Timișoara, Romania
Corporacio Sanitaria Parc Tauli, Sabadell — Barcelona, Spain
Hospital Sant Joan de Deu — Barcelona, Spain
Hospital Universitario 12 de Octubre — Madrid, Spain
Universitario Virgen de la Victoria — Málaga, Spain
Karolinska Universitetssjukhuset Solna — Solna, Sweden
Fulbourn Hospital — Cambridge, England, United Kingdom
Hull Royal Infirmary — Hull, England, United Kingdom
Ninewells Hospital - PPDS — Dundee, Scotland, United Kingdom
Royal Hospital for Children and Young People — Edinburgh, Scotland, United Kingdom

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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