A proof-of-concept study of pitolisant for excessive daytime sleepiness in patients with Prader-Willi syndrome.

ABSTRACT:

Study objectives

The majority of patients with Prader-Willi syndrome experience excessive daytime sleepiness (EDS). This study evaluated the effects of pitolisant, a histamine 3 (H₃)-receptor antagonist/inverse agonist that promotes wakefulness, in patients with Prader-Willi syndrome and EDS.

Methods

In this phase 2, randomized, double-blind, placebo-controlled, proof-of-concept study, patients ages 6-65 years with a confirmed diagnosis of Prader-Willi syndrome with EDS were randomized 1:1:1 to receive lower-dose pitolisant (children/adolescents/adults, 8.9/13.35/17.8 mg), higher-dose pitolisant (children/adolescents/adults, 17.8/26.7/35.6 mg), or matching placebo for 11 weeks (3-week titration/8-week maintenance). The primary endpoint was change from baseline to

SUBMITTER: Revana A 

PROVIDER: S-EPMC12582198 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature