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
Status: Recruiting
Location: See all (57) locations...
Intervention Type: Other, Drug
Study Type: Interventional
Study Phase: Phase 3
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
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:
• 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.
Locations
United States
California
Santa Monica Clinical Trials
RECRUITING
Los Angeles
Center of Excellence in Diabetes and Endocrinology
RECRUITING
Sacramento
Rady Children's Hospital - Scan Diego
RECRUITING
San Diego
Tri-Valley Sleep Center
RECRUITING
San Ramon
Colorado
Colorado Children's Hospital
RECRUITING
Aurora
Delaware
Nemours Children's Hospital
RECRUITING
Wilmington
Georgia
Atlanta Diabetes Associates
WITHDRAWN
Atlanta
Emory University School of Medicine
RECRUITING
Atlanta
Rare Disease Research
WITHDRAWN
Atlanta
Illinois
Ann And Robert H Lurie Children's Hospital of Chicago
RECRUITING
Chicago
Indiana
Riley Children's Hospital
RECRUITING
Indianapolis
Maryland
Johns Hopkins Hospital
RECRUITING
Baltimore
Minnesota
Mayo Clinic-PPDS
RECRUITING
Rochester
North Carolina
Science 37 (at-home option)
RECRUITING
Morrisville
New York
Maimonides Medical Center
RECRUITING
Brooklyn
Montefiore Medical Center
RECRUITING
The Bronx
Ohio
CTI Clinical Research Center
WITHDRAWN
Cincinnati
Center for Human Genetics
RECRUITING
Cleveland
Texas
Texas Children's Hospital
RECRUITING
Houston
Road Runner Research
RECRUITING
San Antonio
Texas Valley Clinical Research
TERMINATED
Weslaco
Wisconsin
Childrens Hospital of Wisconsin
RECRUITING
Milwaukee
Other Locations
Australia
Queensland Children's Hospital
RECRUITING
Brisbane
Royal Prince Alfred Hospital
RECRUITING
Camperdown
Sydney Children's Hospital
RECRUITING
Randwick
Children's Hospital at Westmead
RECRUITING
Westmead
Belgium
UZ Brussels
RECRUITING
Jette
Canada
AMNDX Inc.
RECRUITING
Thornhill
Jodha Tishon Inc.
RECRUITING
Toronto
Denmark
Aarhus University Hospital
RECRUITING
Aarhus
Rigshospitalet
RECRUITING
Copenhagen
France
CHU d'Angers
RECRUITING
Angers
CHU de Toulouse-Hôpital Des Enfants
RECRUITING
Toulouse
Germany
University Hospital Essen
RECRUITING
Essen
Italy
Azienda Ospedaliero Universitaria A Meyer
RECRUITING
Florence
Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN
RECRUITING
Genova
Ospedale San Raffaele S.r.l. - PPDS
RECRUITING
Milan
Azienda Ospedaliero Universitaria Di Modena Policlinico
NOT_YET_RECRUITING
Modena
Azienda Ospedale Università Padova - Dipartimento Salute della Donna e del Bambino - INCIPIT - PIN
RECRUITING
Padua
Ospedale Pediatrico Bambino Gesù IRCCS
NOT_YET_RECRUITING
Roma
IRCCS Materno Infantile Burlo Garofolo - INCIPIT - PIN
RECRUITING
Trieste
Poland
Samodzielny Publiczny Szpital Kliniczny
RECRUITING
Szczecin
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu-Chalubinskiego 2-2a
RECRUITING
Wroclaw
Romania
Institutul National de Endocrinologie C. I. Parhon
RECRUITING
Bucharest
Institutul National de Endocrinologie C. I. Parhon
RECRUITING
Bucharest
National Clinical Center for Children Neurorehabilitation Dr. Nicolae Robanescu
RECRUITING
Bucharest
Louis Turcanu Emergency Clinical Hospital for Children
RECRUITING
Timișoara
Spain
Hospital Sant Joan de Deu
RECRUITING
Barcelona
Hospital Universitario de Fuenlabrada
WITHDRAWN
Fuenlabrada
Hospital Universitario 12 de Octubre
RECRUITING
Madrid
Universitario Virgen de la Victoria
RECRUITING
Málaga
Corporacio Sanitaria Parc Tauli
RECRUITING
Sabadell
Sweden
Karolinska Universitetssjukhuset Solna
RECRUITING
Solna
United Kingdom
Fulbourn Hospital
RECRUITING
Cambridge
Ninewells Hospital - PPDS
RECRUITING
Dundee
Royal Hospital for Children and Young People
NOT_YET_RECRUITING
Edinburgh
Hull Royal Infirmary
RECRUITING
Hull
Contact Information
Primary
Ann Adee
clinicaltrials@harmonybiosciences.com
773-383-6258
Backup
Linnea Ryan
clinicaltrials@harmonybiosciences.com
Time Frame
Start Date: 2024-05-28
Estimated Completion Date: 2027-07
Participants
Target number of participants: 134
Treatments
Experimental: Double-Blind Treatment Period Pitolisant
Pitolisant tablets administered once daily in the morning upon wakening
Placebo_comparator: Double-Blind Treatment Period Placebo
Matching placebo administered tablets once daily in the morning upon wakening
Other: Open-Label Extension Period Pitolisant
Pitolisant tablets administered once daily in the morning upon wakening
Related Therapeutic Areas
Sponsors
Leads: Harmony Biosciences Management, Inc.