A Study of Pitolisant in Patients With Prader-Willi Syndrome
Recruiting · NCT06366464 · Interventional (participants receive a specific treatment) · Lead sponsor: Harmony Biosciences Management, Inc.
View the official record on ClinicalTrials.gov →Interventions studied
Pitolisant tabletPlacebo tablet
What this trial is about
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
Who can take part
Age range
6 Years and older
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 3
Study type
Interventional (participants receive a specific treatment)
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
Where it is running
57 locations listed across 13 US states.
- 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
- Aarhus University Hospital - Aarhus, Denmark
- CHU d'Angers - Angers, France
- Corporacio Sanitaria Parc Tauli, Sabadell - Barcelona, Spain
- Hospital Sant Joan de Deu - Barcelona, Spain
- 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
- Royal Prince Alfred Hospital - Camperdown, Australia
- Rigshospitalet - Copenhagen, Denmark
- University Hospital Essen - Essen, Germany
- Azienda Ospedaliero Universitaria A Meyer - Florence, Italy
- Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN - Genova, Italy
- UZ Brussels - Jette, Belgium
- Hospital Universitario 12 de Octubre - Madrid, Spain
- Ospedale San Raffaele S.r.l. - PPDS - Milan, Italy
- Universitario Virgen de la Victoria - Málaga, Spain
- Azienda Ospedale Università Padova - Dipartimento Salute della Donna e del Bambino - INCIPIT - PIN - Padova, Italy
- Sydney Children's Hospital - Randwick, Australia
- Ospedale Pediatrico Bambino Gesù IRCCS - Roma, Italy
- Karolinska Universitetssjukhuset Solna - Solna, Sweden
- Samodzielny Publiczny Szpital Kliniczny - Szczecin, Poland
- Louis Turcanu Emergency Clinical Hospital for Children - Timișoara, Romania
- CHU de Toulouse-Hôpital Des Enfants - Toulouse, France
- IRCCS Materno Infantile Burlo Garofolo - INCIPIT - PIN - Trieste, Italy
- Children's Hospital at Westmead - Westmead, Australia
- Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu-Chalubinskiego 2-2a - Wroclaw, Poland
- Fulbourn Hospital - Cambridge, England, United Kingdom
- Hull Royal Infirmary - Hull, England, United Kingdom
- AMNDX Inc. - Thornhill, Ontario, Canada
- Jodha Tishon Inc. - Toronto, Ontario, Canada
- Queensland Children's Hospital - Brisbane, Queensland, Australia
- Ninewells Hospital - PPDS - Dundee, Scotland, United Kingdom
- Royal Hospital for Children and Young People - Edinburgh, Scotland, United Kingdom
- University of Florence - Florence, Tuscany, Italy