A Study of Pitolisant in Patients With Prader-Willi Syndrome

NCT06366464 · tracked via the Priya Life Science USA tracker

Sponsor

Harmony Biosciences Management, Inc.

Phase

Phase 3

Started

2024-05-28

Last updated

2026-08-25

Condition(s) studied

Prader-Willi Syndrome

Investigational drug(s) / intervention(s)

Pitolisant tabletPlacebo tablet

Pitolisant tablet: Pitolisant tablet

Placebo tablet: Placebo tablet

Study 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

Eligibility

Sex

ALL

Min age

6 Years

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score — 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.

Trial sites (57)

Facility	City	Region	Status
Santa Monica Clinical Trials	Los Angeles	California	Recruiting
Center of Excellence in Diabetes and Endocrinology	Sacramento	California	Recruiting
Rady Children's Hospital - Scan Diego	San Diego	California	Recruiting
Colorado Children's Hospital	Aurora	Colorado	Recruiting
Nemours Children's Hospital	Wilmington	Delaware	Recruiting
Atlanta Diabetes Associates	Atlanta	Georgia	Withdrawn
Emory University School of Medicine	Atlanta	Georgia	Recruiting
Rare Disease Research	Atlanta	Georgia	Withdrawn
Ann And Robert H Lurie Children's Hospital of Chicago	Chicago	Illinois	Recruiting
Riley Children's Hospital	Indianapolis	Indiana	Recruiting
Johns Hopkins University Hospital	Baltimore	Maryland	Recruiting
Johns Hopkins Hospital	Baltimore	Maryland	Recruiting
Mayo Clinic-PPDS	Rochester	Minnesota	Recruiting
Maimonides Medical Center	Brooklyn	New York	Recruiting
Montefiore Medical Center	The Bronx	New York	Recruiting
Science 37 (at-home option)	Morrisville	North Carolina	Recruiting
Science 37	Morrisville	North Carolina	Recruiting
CTI Clinical Research Center	Cincinnati	Ohio	Withdrawn
Center for Human Genetics	Cleveland	Ohio	Recruiting
Texas Children's Hospital	Houston	Texas	Recruiting
Road Runner Research	San Antonio	Texas	Recruiting
Texas Valley Clinical Research	Weslaco	Texas	Terminated
Childrens Hospital of Wisconsin	Milwaukee	Wisconsin	Recruiting
Queensland Children's Hospital	Brisbane	Queensland	Recruiting
Royal Prince Alfred Hospital	Camperdown	Australia	Recruiting
Sydney Children's Hospital	Randwick	Australia	Recruiting
Children's Hospital at Westmead	Westmead	Australia	Recruiting
UZ Brussels	Jette	Belgium	Recruiting
AMNDX Inc.	Thornhill	Ontario	Recruiting
Jodha Tishon Inc.	Toronto	Ontario	Withdrawn
Aarhus University Hospital	Aarhus	Denmark	Recruiting
Rigshospitalet	Copenhagen	Denmark	Recruiting
CHU d'Angers	Angers	France	Recruiting
CHU de Toulouse-Hôpital Des Enfants	Toulouse	France	Recruiting
University Hospital Essen	Essen	Germany	Recruiting
University of Florence	Florence	Tuscany	Recruiting
Azienda Ospedaliero Universitaria A Meyer	Florence	Italy	Recruiting
Istituto G Gaslini Ospedale Pediatrico IRCCS - INCIPIT - PIN	Genova	Italy	Recruiting
Ospedale San Raffaele S.r.l. - PPDS	Milan	Italy	Recruiting
Azienda Ospedale Università Padova - Dipartimento Salute della Donna e del Bambino - INCIPIT - PIN	Padova	Italy	Recruiting

+ 17 more sites — see the full list on the official registry below.

More Harmony Biosciences Management, Inc. trials in the USA

A Phase 3 Efficacy and Safety Study of HBS-301 in Participants With Idiopathic Hypersomnia (IH) Recruiting

A Phase 3 Efficacy and Safety Study of HBS-301 in Participants With Narcolepsy Recruiting

A Study of Pitolisant in Participants With Prader-Willi Syndrome Enrolling by invitation

WAKIX® (Pitolisant) Pregnancy Registry Recruiting

Other trials for the same condition

A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment Recruiting

A Study of Setmelanotide in Patients With Prader-Willi Syndrome Active, not recruiting

Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome Enrolling by invitation

A Study of CSTI-500 in Patients With Prader-Willi Syndrome Recruiting

Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06366464 on ClinicalTrials.gov ↗ ← All trials in the USA