NCT06366464

A Study of Pitolisant in Patients With Prader-Willi Syndrome

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 134 (estimated)
• Sponsor: Harmony Biosciences Management, Inc. · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

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

Detailed description

The study will consist of an up to 45-day Screening/Baseline Period, a Double-Blind Treatment Period, and an optional Open-Label Extension Period. After completion of all Baseline assessments, patients who meet all eligibility criteria will be randomized 1:1 to receive once daily pitolisant or matching placebo. During the Double-Blind Treatment Period, in-person visits will be at Day 29, Day 57, and Day 77. Patients who do not elect to enter the Open-Label Extension Period will have follow-up visits 15 days and 30 days after the final dose of study drug. During the optional Open-Label Extension Period, in-person visits will be at Day 113, Day 260, and Day 441. Patients will have follow-up visits 15 days and 30 days after the final dose of pitolisant.

Conditions

• Prader-Willi Syndrome

Interventions

Type	Name	Description
DRUG	Pitolisant tablet	Pitolisant tablet
OTHER	Placebo tablet	Placebo tablet

Timeline

• Start date: 2024-05-28
• Primary completion: 2026-07-01
• Completion: 2027-07-01
• First posted: 2024-04-16
• Last updated: 2026-03-17

Locations

54 sites across 13 countries: United States, Australia, Belgium, Canada, Denmark, France, Germany, Italy, Poland, Romania, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study