Recruiting
Phase 3

Pitolisant

Sponsor:

Harmony Biosciences Management, Inc.

Code:

NCT06366464

Conditions

Prader-Willi Syndrome

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Interventions

Pitolisant tablet

Placebo tablet

Study Details

Brief summary:

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

Conditions

Prader-Willi Syndrome

Study ID

NCT06366464

Start date

May 28, 2024

Status verified date

Aug, 2026

Completion date

Apr, 2028

Anticipated

Primary completion date

Apr, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

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

Enrollment

134 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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

Interventions

Pitolisant tablet

Pitolisant tablet

Placebo tablet

Placebo tablet

Primary outcome measure

  • 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) ]

Central Contacts and Locations

Central contacts

Ann Adee

773-383-6258clinicaltrials@harmonybiosciences.com

Linnea Ryan

clinicaltrials@harmonybiosciences.com

Locations

Santa Monica Clinical Trials

Recruiting

Los Angeles, California, United States, 90025

Contacts

Samantha Montenez

310-586-0843e. smontanez@smclinicaltrials.com

Principal Investigator:

Daniel Norman, MD

Center of Excellence in Diabetes and Endocrinology

Recruiting

Sacramento, California, United States, 95821-2123

Contacts

Natalie Marlen

279-210-9814marlenn@sutterhealth.org

Principal Investigator:

Gnanagurudasan Prakasam, MD

Rady Children's Hospital - Scan Diego

Recruiting

San Diego, California, United States, 92123

Contacts

Megan Warner

mwarner2@rchsd.org

Principal Investigator:

Rakesh Bhattacharjee, MD

Colorado Children's Hospital

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Nathan Holly

920-264-4742nathan.holly@childrenscolorado.org

Principal Investigator:

Shawn McCandless, MD

Nemours Children's Hospital

Recruiting

Wilmington, Delaware, United States, 19803-3607

Contacts

Kimberly Renner

302-651-6400kimberly.renner@nemours.org

Principal Investigator:

Aaron Chidekel, MD

Emory University School of Medicine

Recruiting

Atlanta, Georgia, United States, 30322

Contacts

Gwendolyn Gunn

404-778-8552gwendolyn.gisiner.gunn@emory.edu

Principal Investigator:

Michael Gambello, MD, PhD

Ann And Robert H Lurie Children's Hospital of Chicago

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Sarayu Ratnam, PhD

312-227-6617sratnam@luriechildrens.org

Principal Investigator:

Priya Khanna, MD

Riley Children's Hospital

Recruiting

Indianapolis, Indiana, United States, 46202

Contacts

Julia Meyer

317-278-7118meyerj@iupui.edu

Principal Investigator:

Hasnaa Jalou, MD

Johns Hopkins University Hospital

Recruiting

Baltimore, Maryland, United States, 21205

Principal Investigator:

Anne Scheimann, MD, MBA

Johns Hopkins Hospital

Recruiting

Baltimore, Maryland, United States, 21224

Contacts

Ann Scheimann

410-955-8769ascheima@jhmi.edu,

Principal Investigator:

Ann Scheimann, MD

Mayo Clinic-PPDS

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Brittany Kehoe

507-293-2316kehoe.brittany@mayo.edu

Principal Investigator:

Queenie Tan, MD

Maimonides Medical Center

Recruiting

Brooklyn, New York, United States, 11219

Contacts

Hasan Mustafic

718-283-8170HMustafic@maimo.org,

Principal Investigator:

Edward Yi, MD

Montefiore Medical Center

Recruiting

The Bronx, New York, United States, 10467

Contacts

Eric Hollander, MD

eholland@montefiore.org

Principal Investigator:

Eric Hollander, MD

Science 37 (at-home option)

Recruiting

Morrisville, North Carolina, United States, 27560

Contacts

Dr. Sara Trepanier

310-929-8473HarmonyPWS@science37.com

Principal Investigator:

Dr. Sara Trepanier

Science 37

Recruiting

Morrisville, North Carolina, United States, 27560

Principal Investigator:

Sara Trepanier, MD

Center for Human Genetics

Recruiting

Cleveland, Ohio, United States, 441016

Contacts

Mehjabeen Hossain

216-869-9202mehjabeen.hossain@uhhospitals.org

Principal Investigator:

Anna Mitchell, MD

Texas Children's Hospital

Recruiting

Houston, Texas, United States, 77030

Contacts

Paushpala Sen

832-822-1343pxsen3@texaschildrens.org

Ifeoma Ibekwe

icibekwe@texaschildrens.org

Principal Investigator:

Amee Revana, DO

Road Runner Research

Recruiting

San Antonio, Texas, United States, 78249-3539

Contacts

Nancy Lopez

210-949-0505nlopez@rrresearchsa.com

Principal Investigator:

Tarak Patel, MD

Childrens Hospital of Wisconsin

Recruiting

Milwaukee, Wisconsin, United States, 53226

Contacts

Paula Engelking

414-266-3289pengelki@mcw.edu

Principal Investigator:

Donald Basel, MD

AMNDX Inc.

Recruiting

Thornhill, Ontario, Canada

Contacts

Urszula Lamoureux

416-435-6244urszula.research@gmail.com

Principal Investigator:

Alan Lowe, MD

More Information

Sponsor

Harmony Biosciences Management, Inc.

Last update posted

Aug 25, 2026

Last verified

Aug, 2026

Keywords

  • pitolisant
  • excessive daytime sleepiness
  • irritable and disruptive behaviors
  • Prader-Willi syndrome

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Harmony Biosciences Management, Inc. on 2026-08-25.