A Study of Pitolisant in Patients with Prader-Willi Syndrome

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

Conditions

Prader-Willi Syndrome

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Study Overview

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Study Details

Study overview

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

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

A Study of Pitolisant in Patients with Prader-Willi Syndrome

Condition
Prader-Willi Syndrome
Intervention / Treatment

-

Contacts and Locations

Los Angeles

Santa Monica Clinical Trials, Los Angeles, California, United States, 90025

Sacramento

Center of Excellence in Diabetes and Endocrinology, Sacramento, California, United States, 95821-2123

San Diego

Rady Children's Hospital - Scan Diego, San Diego, California, United States, 92123

San Ramon

Tri-Valley Sleep Center, San Ramon, California, United States, 94583

Aurora

Colorado Children's Hospital, Aurora, Colorado, United States, 80045

Wilmington

Nemours Children's Hospital, Wilmington, Delaware, United States, 19803-3607

Atlanta

Atlanta Diabetes Associates, Atlanta, Georgia, United States, 30318

Atlanta

Rare Disease Research, Atlanta, Georgia, United States, 30329

Indianapolis

Riley Children's Hospital, Indianapolis, Indiana, United States, 46202

Baltimore

Johns Hopkins Hospital, Baltimore, Maryland, United States, 21224

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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

Ages Eligible for Study

6 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Harmony Biosciences, LLC,

Study Record Dates

2027-07