Clinical Trial

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Recruiting Phase 3
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Record status
This record was last updated March 17, 2026 (before its estimated July 2026 completion). Its status may not reflect the trial's current state.
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
Protocol Amendment History 15 amendments
This ClinicalTrials.gov record has been amended 15 times since 2024-04-12; most recent amendment 2026-03-13.
Trial Details
NCT Number NCT06366464
Lead Sponsor Harmony Biosciences Management, Inc.
Conditions Prader-Willi Syndrome
Enrollment 134 participants
Start Date 2024-05-28
Primary Completion 2026-07 (estimated)
Study Completion 2027-07 (estimated)
Updated on ClinicalTrials.gov 2026-03-17