Skip to content
Bored Studies
RecruitingPhase 3

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Harmony Biosciences Management, Inc. · NCT06366464

View Study & Apply (opens in a new tab)

Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 6+ · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 3
Target enrollment
134
US sites recruiting
19
Study start
May 28, 2024
Record last updated
August 25, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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

Being studied: Pitolisant tablet; Placebo tablet

Conditions: Prader-Willi Syndrome

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

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

Recruiting US locations

  • Santa Monica Clinical Trials

    Los Angeles, CA 90025

    Site contact: Samantha Montenez · 310-586-0843 · e. smontanez@smclinicaltrials.com

  • Center of Excellence in Diabetes and Endocrinology

    Sacramento, CA 95821-2123

    Site contact: Natalie Marlen · 279-210-9814 · marlenn@sutterhealth.org

  • Rady Children's Hospital - Scan Diego

    San Diego, CA 92123

    Site contact: Megan Warner · mwarner2@rchsd.org

  • Colorado Children's Hospital

    Aurora, CO 80045

    Site contact: Nathan Holly · 920-264-4742 · nathan.holly@childrenscolorado.org

  • Nemours Children's Hospital

    Wilmington, DE 19803-3607

    Site contact: Kimberly Renner · 302-651-6400 · kimberly.renner@nemours.org

  • Emory University School of Medicine

    Atlanta, GA 30322

    Site contact: Gwendolyn Gunn · 404-778-8552 · gwendolyn.gisiner.gunn@emory.edu

  • Ann And Robert H Lurie Children's Hospital of Chicago

    Chicago, IL 60611

    Site contact: Sarayu Ratnam, PhD · 312-227-6617 · sratnam@luriechildrens.org

  • Riley Children's Hospital

    Indianapolis, IN 46202

    Site contact: Julia Meyer · 317-278-7118 · meyerj@iupui.edu

  • Johns Hopkins University Hospital

    Baltimore, MD 21205

    Site contact: Anne Scheimann, MD, MBA

  • Johns Hopkins Hospital

    Baltimore, MD 21224

    Site contact: Ann Scheimann · 410-955-8769 · ascheima@jhmi.edu,

  • Mayo Clinic-PPDS

    Rochester, MN 55905

    Site contact: Brittany Kehoe · 507-293-2316 · kehoe.brittany@mayo.edu

  • Maimonides Medical Center

    Brooklyn, NY 11219

    Site contact: Hasan Mustafic · 718-283-8170 · HMustafic@maimo.org,

  • Montefiore Medical Center

    The Bronx, NY 10467

    Site contact: Eric Hollander, MD · eholland@montefiore.org

  • Science 37 (at-home option)

    Morrisville, NC 27560

    Site contact: Dr. Sara Trepanier · 310-929-8473 · HarmonyPWS@science37.com

  • Science 37

    Morrisville, NC 27560

    Site contact: Sara Trepanier, MD

  • Center for Human Genetics

    Cleveland, OH 441016

    Site contact: Mehjabeen Hossain · 216-869-9202 · mehjabeen.hossain@uhhospitals.org

  • Texas Children's Hospital

    Houston, TX 77030

    Site contact: Paushpala Sen · 832-822-1343 · pxsen3@texaschildrens.org

  • Road Runner Research

    San Antonio, TX 78249-3539

    Site contact: Nancy Lopez · 210-949-0505 · nlopez@rrresearchsa.com

  • Childrens Hospital of Wisconsin

    Milwaukee, WI 53226

    Site contact: Paula Engelking · 414-266-3289 · pengelki@mcw.edu

Study contacts (from the official record)

  • Ann Adee · 773-383-6258 · clinicaltrials@harmonybiosciences.com
  • Linnea Ryan · clinicaltrials@harmonybiosciences.com
All sleep studies in California →
A Study of Pitolisant in Patients With Prader-Willi Syndrome (NCT06366464) | Bored Studies