A Study of Pitolisant in Patients With Prader-Willi Syndrome
Recruiting now · Phase 3 · Has a placebo group
Conditions studied: Prader-Willi Syndrome
In brief
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
Key facts
- Study ID
- NCT06366464
- Run by
- Harmony Biosciences Management, Inc.
- People needed
- 134
- Starts
- 2024-05-28
- Expected to finish
- 2027-07-01
- Last updated by the study team
- 2026-03-17
Who can join
Age: 6 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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 it is running
- Jodha Tishon Inc. — Toronto, Ontario, Canada (enrolling)
- Santa Monica Clinical Trials — Los Angeles, California, United States (enrolling)
- UZ Brussels — Jette, Belgium (enrolling)
- AMNDX Inc. — Thornhill, Ontario, Canada (enrolling)
- Rady Children's Hospital - Scan Diego — San Diego, California, United States (enrolling)
- Emory University School of Medicine — Atlanta, Georgia, United States (enrolling)
- Center of Excellence in Diabetes and Endocrinology — Sacramento, California, United States (enrolling)
- Ann And Robert H Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
- Nemours Children's Hospital — Wilmington, Delaware, United States (enrolling)
- Johns Hopkins Hospital — Baltimore, Maryland, United States (enrolling)
- Mayo Clinic-PPDS — Rochester, Minnesota, United States (enrolling)
- Maimonides Medical Center — Brooklyn, New York, United States (enrolling)
- Montefiore Medical Center — The Bronx, New York, United States (enrolling)
- Science 37 (at-home option) — Morrisville, North Carolina, United States (enrolling)
- Colorado Children's Hospital — Aurora, Colorado, United States (enrolling)
- Center for Human Genetics — Cleveland, Ohio, United States (enrolling)
- Texas Children's Hospital — Houston, Texas, United States (enrolling)
- Road Runner Research — San Antonio, Texas, United States (enrolling)
- Riley Children's Hospital — Indianapolis, Indiana, United States (enrolling)
- Childrens Hospital of Wisconsin — Milwaukee, Wisconsin, United States (enrolling)
- Queensland Children's Hospital — Brisbane, Queensland, Australia (enrolling)
- Royal Prince Alfred Hospital — Camperdown, Australia (enrolling)
- Sydney Children's Hospital — Randwick, Australia (enrolling)
- Children's Hospital at Westmead — Westmead, Australia (enrolling)
- Aarhus University Hospital — Aarhus, Denmark (enrolling)
Full record on ClinicalTrials.gov
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