NCT06366464 · Harmony Biosciences Management, Inc.
A Study of Pitolisant in Patients With Prader-Willi Syndrome
What this study is about
This is a Phase 3, randomly assigned, where neither patients nor doctors know which treatment is given, compared against an inactive treatment, conducted at multiple hospitals, global clinical study to assess the effectiveness and safety of pitolisant in patients living with Prader-Willi syndrome.
View original scientific description
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.
Interventions
DRUG
Pitolisant tablet
Pitolisant tablet
OTHER
Placebo tablet
Placebo tablet
Primary outcome measures
Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score
Time frame: Baseline and end of the Double Blind Treatment Period (Day 77)
The PROMIS-SRI item bank consists of 13 items with a 5-point rating scale.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
Where
- Los Angeles, California
- Sacramento, California
- San Diego, California
- Aurora, Colorado
- Wilmington, Delaware
- Atlanta, Georgia
- Chicago, Illinois
- Indianapolis, Indiana
- Baltimore, Maryland
- Rochester, Minnesota
- Brooklyn, New York
- The Bronx, New York
And 7 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 17, 2026 · Source of record for eligibility and locations