RecruitingPrader-Willi Syndrome

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Eligible age

6+ yrs

Accepts

All genders

Locations

13 states

Healthy volunteers

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About this study

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

Sponsor: Harmony Biosciences Management, Inc.

You may qualify if…

✓ 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.

You may not qualify if…

✕ 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