A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension

Phase 2 Completed 65 enrolled Harmony Biosciences Management, Inc.

RandomizedParallel-groupTriple-blindTreatment

Summary

The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Prader Willi syndrome (PWS) ages 6 to 65 years.

Timeline

Start: 2020-12-09
Primary completion: 2022-08-17
Completion: 2026-01-21

Publications

Revana A, Bhattacharjee R, Miller JL, Chidekel A, Khanna P, Ratnam S, Runyan G, Bauer E, Davis Rapchak K, Seiden D, Budur K, Dayno JM. A proof-of-concept study of pitolisant for excessive daytime sleepiness in patients with Prader-Willi syndrome. J Clin Sleep Med. 2025 Nov 1;21(11):1893-1902. doi: 10.5664/jcsm.11800.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Pitolisant	Small molecule	8.9 mg	Oral
Subject	Pitolisant	Small molecule	13.35 mg	Oral
Subject	Pitolisant	Small molecule	17.8 mg	Oral
Subject	Pitolisant	Small molecule	26.7 mg	Oral
Subject	Pitolisant	Small molecule	35.6 mg	Oral

A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension

Summary

Timeline

Publications

Drugs

Indications