TEMPO
Research type
Research Study
Full title
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
IRAS ID
1010453
Contact name
Michele Roy
Contact email
Sponsor organisation
Harmony Biosciences Management, Inc.
Clinicaltrials.gov Identifier
Research summary
Prader-Willi syndrome (PWS) is a rare genetic disorder that results in several physical, mental, and behavioural problems. This is a Phase 3, randomised, double-blind, placebo-controlled, multicentre, global clinical study sponsored by Harmony Biosciences to test a drug called pitolisant in people with PWS.
Pitolisant is approved, by the United States Food and Drug Administration (FDA), and is available by prescription in the US for the treatment of excessive daytime sleepiness (EDS) or cataplexy (sudden muscle weakness while awake) in adults with a sleep disorder called narcolepsy and for the treatment of EDS in children 6 years of age and older with narcolepsy. In the European Union (EU) and other countries outside of the US, pitolisant is approved for treating a sleep disorder called narcolepsy, with or without cataplexy (sudden muscle weakness while awake), in adults, adolescents, and children ages 6 years and above. Pitolisant is not approved for treating the symptoms of PWS.
The purpose of this study is to test pitolisant in people with PWS to find out how safe it is, how effective it is in treating excessive daytime sleepiness and, how it affects participants’ behaviour and hunger. The study will also test to check how much of the study drug is in the participant’s body.
Approximately 134 participants ages 6 years and above will be randomised at about 65 study sites globally. Participants will be randomised 1:1 to receive pitolisant or matching placebo and randomisation will be stratified by age group and geographic region. The study will consist of a Screening/Baseline Period (up to 45 days), a Double-Blind Treatment Period (77 days), and an optional Open-Label Extension Period (364 days) and 30 days of safety follow-up.
REC name
Wales REC 3
REC reference
24/WA/0369
Date of REC Opinion
24 Feb 2025
REC opinion
Further Information Favourable Opinion