Phase 3 Study of Pitolisant to Treat Excessive Daytime Sleepiness in Patients 6 Years and Older With Prader-Willi Syndrome Includes Double-Blind and Open-Label Extension Phases

What this Trial Is About

Researchers are evaluating the effectiveness and safety of pitolisant in treating excessive daytime sleepiness EDS in patients aged 6 years and older with Prader-Willi syndrome. This Phase 3, randomized, double-blind, placebo-controlled, global study also aims to assess how pitolisant affects irritable and disruptive behaviors, hyperphagia, and other behavioral problems such as social withdrawal, stereotypic behavior, hyperactivity, noncompliance, and inappropriate speech. The study includes up to a 45-day screening and baseline period followed by a double-blind treatment phase where participants are randomly assigned to receive either pitolisant tablets or placebo once daily in the morning. In-person visits occur on Days 29, 57, and 77 during this period. Afterward, participants may choose to enter an optional open-label extension period with pitolisant, which includes visits on Days 113, 260, and 441. Follow-up visits are scheduled 15 and 30 days after the final dose in both the double-blind and extension phases. Participants will be closely monitored through various assessments including patient-reported sleep impairment scales, caregiver and clinical impressions of sleepiness and behavior, and questionnaires measuring hyperphagia and other behavioral issues. Safety is monitored by tracking treatment-emergent adverse events throughout the study. The total participation duration may extend over a year for those in the open-label extension, with multiple visits and follow-ups to evaluate the study outcomes comprehensively.

A Study of Pitolisant in Patients With Prader-Willi Syndrome