A Study of Pitolisant in Patients With Prader-Willi Syndrome
NCT ID: NCT06366464
Last Updated: 2025-12-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
134 participants
INTERVENTIONAL
2024-05-28
2027-07-31
Brief Summary
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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
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Detailed Description
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After completion of all Baseline assessments, patients who meet all eligibility criteria will be randomized 1:1 to receive once daily pitolisant or matching placebo. During the Double-Blind Treatment Period, in-person visits will be at Day 29, Day 57, and Day 77. Patients who do not elect to enter the Open-Label Extension Period will have follow-up visits 15 days and 30 days after the final dose of study drug.
During the optional Open-Label Extension Period, in-person visits will be at Day 113, Day 260, and Day 441. Patients will have follow-up visits 15 days and 30 days after the final dose of pitolisant.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Double-Blind Treatment Period Pitolisant
Pitolisant tablets administered once daily in the morning upon wakening
Pitolisant tablet
Pitolisant tablet
Double-Blind Treatment Period Placebo
Matching placebo administered tablets once daily in the morning upon wakening
Placebo tablet
Placebo tablet
Open-Label Extension Period Pitolisant
Pitolisant tablets administered once daily in the morning upon wakening
Pitolisant tablet
Pitolisant tablet
Interventions
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Pitolisant tablet
Pitolisant tablet
Placebo tablet
Placebo tablet
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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
6 Years
ALL
No
Sponsors
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Harmony Biosciences Management, Inc.
INDUSTRY
Responsible Party
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Locations
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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
Tri-Valley Sleep Center
San Ramon, 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
Padua, , 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 Universitario de Fuenlabrada
Fuenlabrada, Madrid, 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
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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HBS-101-CL-312
Identifier Type: -
Identifier Source: org_study_id
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