Actively Recruiting
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
Led by Harmony Biosciences Management, Inc. · Updated on 2026-03-17
134
Participants Needed
54
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effectiveness and safety of pitolisant in treating excessive daytime sleepiness (EDS) in patients aged 6 years and older with Prader-Willi syndrome. This Phase 3, randomized, double-blind, placebo-controlled, global study also aims to assess how pitolisant affects irritable and disruptive behaviors, hyperphagia, and other behavioral problems such as social withdrawal, stereotypic behavior, hyperactivity, noncompliance, and inappropriate speech. The study includes up to a 45-day screening and baseline period followed by a double-blind treatment phase where participants are randomly assigned to receive either pitolisant tablets or placebo once daily in the morning. In-person visits occur on Days 29, 57, and 77 during this period. Afterward, participants may choose to enter an optional open-label extension period with pitolisant, which includes visits on Days 113, 260, and 441. Follow-up visits are scheduled 15 and 30 days after the final dose in both the double-blind and extension phases. Participants will be closely monitored through various assessments including patient-reported sleep impairment scales, caregiver and clinical impressions of sleepiness and behavior, and questionnaires measuring hyperphagia and other behavioral issues. Safety is monitored by tracking treatment-emergent adverse events throughout the study. The total participation duration may extend over a year for those in the open-label extension, with multiple visits and follow-ups to evaluate the study outcomes comprehensively.
CONDITIONS
Brief Title
A Study of Pitolisant in Patients With Prader-Willi Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Genetically confirmed diagnosis of Prader-Willi syndrome
- Excessive daytime sleepiness
- Has a consistent parent or caregiver willing and able to complete study assessments
- Patient or caregiver is capable of understanding and following study requirements and taking oral study medication
You will not qualify if you...
- Diagnosis of sleep apnea that is not adequately controlled
- Diagnosis of hypersomnia caused by another sleep or medical disorder
- Participation in another interventional study with investigational treatment within 30 days or 5 half-lives before screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - Up to 45 days
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - Approximately 11 weeks
Participants receive either pitolisant or placebo tablets once daily in the morning upon wakening during the double-blind treatment period.
In-person visits at Day 29, Day 57, and Day 77
Duration - 30 days after final dose
Participants who do not enter the extension period have follow-up assessments after the final dose of study drug.
2 visits at 15 days and 30 days after final dose
Duration - Up to approximately 1 year and 3 months
Participants in the open-label extension period receive pitolisant tablets once daily in the morning upon wakening.
In-person visits at Day 113, Day 260, and Day 441
Duration - 30 days after final dose
Participants in the open-label extension period have follow-up assessments after the final dose of pitolisant.
2 visits at 15 days and 30 days after final dose
Trial Site Locations
Total: 54 locations
1
Santa Monica Clinical Trials
Los Angeles, California, United States, 90025
Actively Recruiting
2
Center of Excellence in Diabetes and Endocrinology
Sacramento, California, United States, 95821-2123
Actively Recruiting
3
Rady Children's Hospital - Scan Diego
San Diego, California, United States, 92123
Actively Recruiting
4
Colorado Children's Hospital
Aurora, Colorado, United States, 80045
Actively Recruiting
5
Nemours Children's Hospital
Wilmington, Delaware, United States, 19803-3607
Actively Recruiting
6
Atlanta Diabetes Associates
Atlanta, Georgia, United States, 30318
Withdrawn
7
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Actively Recruiting
8
Rare Disease Research
Atlanta, Georgia, United States, 30329
Withdrawn
9
Ann And Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
10
Riley Children's Hospital
Indianapolis, Indiana, United States, 46202
Actively Recruiting
11
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21224
Actively Recruiting
12
Mayo Clinic-PPDS
Rochester, Minnesota, United States, 55905
Actively Recruiting
13
Maimonides Medical Center
Brooklyn, New York, United States, 11219
Actively Recruiting
14
Montefiore Medical Center
The Bronx, New York, United States, 10467
Actively Recruiting
15
Science 37 (at-home option)
Morrisville, North Carolina, United States, 27560
Actively Recruiting
16
CTI Clinical Research Center
Cincinnati, Ohio, United States, 45212
Withdrawn
17
Center for Human Genetics
Cleveland, Ohio, United States, 441016
Actively Recruiting
18
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
19
Road Runner Research
San Antonio, Texas, United States, 78249-3539
Actively Recruiting
20
Texas Valley Clinical Research
Weslaco, Texas, United States, 78596
Terminated
21
Childrens Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
22
Queensland Children's Hospital
Brisbane, Queensland, Australia
Actively Recruiting
23
Royal Prince Alfred Hospital
Camperdown, Australia
Actively Recruiting
24
Sydney Children's Hospital
Randwick, Australia
Actively Recruiting
25
Children's Hospital at Westmead
Westmead, Australia
Actively Recruiting
26
UZ Brussels
Jette, Belgium
Actively Recruiting
27
AMNDX Inc.
Thornhill, Ontario, Canada
Actively Recruiting
28
Jodha Tishon Inc.
Toronto, Ontario, Canada
Actively Recruiting
29
Aarhus University Hospital
Aarhus, Denmark
Actively Recruiting
30
Rigshospitalet
Copenhagen, Denmark
Actively Recruiting
31
CHU d'Angers
Angers, France
Actively Recruiting
32
CHU de Toulouse-Hôpital Des Enfants
Toulouse, France
Actively Recruiting
33
University Hospital Essen
Essen, Germany
Actively Recruiting
34
Azienda Ospedaliero Universitaria A Meyer
Florence, Italy
Actively Recruiting
35
Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN
Genova, Italy
Actively Recruiting
36
Ospedale San Raffaele S.r.l. - PPDS
Milan, Italy
Actively Recruiting
37
Azienda Ospedale Università Padova - Dipartimento Salute della Donna e del Bambino - INCIPIT - PIN
Padova, Italy
Actively Recruiting
38
Ospedale Pediatrico Bambino Gesù IRCCS
Roma, Italy
Actively Recruiting
39
IRCCS Materno Infantile Burlo Garofolo - INCIPIT - PIN
Trieste, Italy
Actively Recruiting
40
Samodzielny Publiczny Szpital Kliniczny
Szczecin, Poland
Actively Recruiting
41
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu-Chalubinskiego 2-2a
Wroclaw, Poland
Actively Recruiting
42
Institutul National de Endocrinologie C. I. Parhon
Bucharest, Romania
Actively Recruiting
43
Institutul National de Endocrinologie C. I. Parhon
Bucharest, Romania
Actively Recruiting
44
National Clinical Center for Children Neurorehabilitation "Dr. Nicolae Robanescu"
Bucharest, Romania
Actively Recruiting
45
Louis Turcanu Emergency Clinical Hospital for Children
Timișoara, Romania
Actively Recruiting
46
Corporacio Sanitaria Parc Tauli, Sabadell
Barcelona, Spain
Actively Recruiting
47
Hospital Sant Joan de Deu
Barcelona, Spain
Actively Recruiting
48
Hospital Universitario 12 de Octubre
Madrid, Spain
Actively Recruiting
49
Universitario Virgen de la Victoria
Málaga, Spain
Actively Recruiting
50
Karolinska Universitetssjukhuset Solna
Solna, Sweden
Actively Recruiting
51
Fulbourn Hospital
Cambridge, England, United Kingdom
Actively Recruiting
52
Hull Royal Infirmary
Hull, England, United Kingdom
Actively Recruiting
53
Ninewells Hospital - PPDS
Dundee, Scotland, United Kingdom
Actively Recruiting
54
Royal Hospital for Children and Young People
Edinburgh, Scotland, United Kingdom
Actively Recruiting
Research Team
A
Ann Adee
L
Linnea Ryan
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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