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A Study of Pitolisant in Patients With Prader-Willi Syndrome
Sponsor: Harmony Biosciences Management, Inc.
Summary
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
Official title: A Phase 3, Randomized, Double-Blind, Placebo-controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients With Prader-Willi Syndrome
Key Details
Gender
All
Age Range
6 Years - Any
Study Type
INTERVENTIONAL
Enrollment
134
Start Date
2024-05-28
Completion Date
2027-07
Last Updated
2026-03-17
Healthy Volunteers
No
Conditions
Interventions
Pitolisant tablet
Pitolisant tablet
Placebo tablet
Placebo tablet
Locations (54)
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 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
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
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