RECRUITING

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Conditions

Prader-Willi Syndrome pitolisant excessive daytime sleepiness irritable and disruptive behaviors

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

A Phase 3, Randomized, Double-Blind, Placebo-controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients With Prader-Willi Syndrome

Quick Facts

Study Start:2024-05-28

Study Completion:2027-07

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06366464

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	* 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

Inclusion Criteria

Exclusion 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.

* 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

Contacts and Locations

Study Contact

Ann Adee

CONTACT

773-383-6258

clinicaltrials@harmonybiosciences.com

Linnea Ryan

CONTACT

clinicaltrials@harmonybiosciences.com

Study Locations (Sites)

Santa Monica Clinical Trials

Los Angeles, California, 90025

United States

Center of Excellence in Diabetes and Endocrinology

Sacramento, California, 95821-2123

United States

Rady Children's Hospital - Scan Diego

San Diego, California, 92123

United States

Tri-Valley Sleep Center

San Ramon, California, 94583

United States

Colorado Children's Hospital

Aurora, Colorado, 80045

United States

Nemours Children's Hospital

Wilmington, Delaware, 19803-3607

United States

Atlanta Diabetes Associates

Atlanta, Georgia, 30318

United States

Emory University School of Medicine

Atlanta, Georgia, 30322

United States

Rare Disease Research

Atlanta, Georgia, 30329

United States

Ann And Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611

United States

Riley Children's Hospital

Indianapolis, Indiana, 46202

United States

Johns Hopkins Hospital

Baltimore, Maryland, 21224

United States

Mayo Clinic-PPDS

Rochester, Minnesota, 55905

United States

Montefiore Medical Center

Bronx, New York, 10467

United States

Maimonides Medical Center

Brooklyn, New York, 11219

United States

Science 37 (at-home option)

Morrisville, North Carolina, 27560

United States

CTI Clinical Research Center

Cincinnati, Ohio, 45212

United States

Center for Human Genetics

Cleveland, Ohio, 441016

United States

Texas Children's Hospital

Houston, Texas, 77030

United States

Road Runner Research

San Antonio, Texas, 78249-3539

United States

Texas Valley Clinical Research

Weslaco, Texas, 78596

United States

Childrens Hospital of Wisconsin

Milwaukee, Wisconsin, 53226

United States

Collaborators and Investigators

Sponsor: Harmony Biosciences Management, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-05-28

Study Completion Date2027-07

Study Record Updates

Study Start Date2024-05-28

Study Completion Date2027-07

Terms related to this study

Keywords Provided by Researchers

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

Additional Relevant MeSH Terms

Prader-Willi Syndrome