Clinical Trials in the USA / NCT06366464
Recruiting
Phase 3
A Study of Pitolisant in Patients With Prader-Willi Syndrome
NCT06366464 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Prader-Willi Syndrome
Investigational drug(s) / intervention(s)
Pitolisant tablet →Placebo 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
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
Other trials for the same condition
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
Active, not recruiting
A Study of Pitolisant in Participants With Prader-Willi Syndrome
Enrolling by invitation
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome
Enrolling by invitation
A Study of Setmelanotide 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