A Study of Pitolisant in Patients With Prader-Willi Syndrome
Purpose
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
Condition
- Prader-Willi Syndrome
Eligibility
- Eligible Ages
- Over 6 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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 |
|
Recruiting Locations
Los Angeles 5368361, California 5332921 90025
Sacramento 5389489, California 5332921 95821-2123
San Diego 5391811, California 5332921 92123
San Ramon 5392593, California 5332921 94583
Aurora 5412347, Colorado 5417618 80045
Wilmington 4145381, Delaware 4142224 19803-3607
Atlanta 4180439, Georgia 4197000 30322
Chicago 4887398, Illinois 4896861 60611
Indianapolis 4259418, Indiana 4921868 46202
Baltimore 4347778, Maryland 4361885 21224
Rochester 5043473, Minnesota 5037779 55905
Brooklyn 5110302, New York 5128638 11219
The Bronx 5110266, New York 5128638 10467
Morrisville 4480285, North Carolina 4482348 27560
Cleveland 5150529, Ohio 5165418 441016
Houston 4699066, Texas 4736286 77030
San Antonio 4726206, Texas 4736286 78249-3539
Milwaukee 5263045, Wisconsin 5279468 53226
More Details
- Status
- Recruiting
- Sponsor
- Harmony Biosciences Management, Inc.
Detailed Description
The study will consist of an up to 45-day Screening/Baseline Period, a Double-Blind Treatment Period, and an optional Open-Label Extension Period. 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.