NCT07219485

A Study of Pitolisant in Participants With Prader-Willi Syndrome

An Open-Label Study to Evaluate Safety of Pitolisant in Participants With Prader-Willi Syndrome

• Status: Enrolling By Invitation
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 150 (estimated)
• Sponsor: Harmony Biosciences Management, Inc. · Industry
• Sex: All
• Age: 7 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to assess safety of pitolisant in eligible participants with Prader-Willi syndrome (PWS) who completed the End of Treatment (EOT) visit in a parent study (HBS-101-CL-002 \[Open Label Extension (OLE)\], HBS- 101-CL-004, or HBS-101-CL-312 OLE).

Detailed description

This is a Phase 3, open-label study evaluating the long-term safety of pitolisant in approximately 150 participants with Prader-Willi syndrome who have completed participation in qualifying parent studies (HBS-101-CL-002 \[OLE\], HBS-101-CL-004, or HBS-101-CL-312 \[OLE\]). Safety assessments will be conducted every 6 months. This study is open to participants from the qualified parent studies who were enrolled at US sites only.

Conditions

• Prader-Willi Syndrome

Interventions

Type	Name	Description
DRUG	Pitolisant	* Pitolisant 4.45 mg tablets * Pitolisant 17.8 mg tablets

Timeline

• Start date: 2025-08-20
• Primary completion: 2030-08-01
• Completion: 2030-08-01
• First posted: 2025-10-21
• Last updated: 2026-03-17

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study