Study Purpose
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 study objectives are to evaluate the impact of pitolisant on:
- Irritable and disruptive behaviors
- Hyperphagia
- Other behavioral problems including social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech
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.
Approximately 134 patients who meet all eligibility criteria and complete all of the baseline assessments 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.
Learn more about what you can expect should you choose to participate in this 30-minute webinar: Watch the Webinar Now!
Recruitment Criteria
Study Type: Interventional
Eligible Ages: 6-65 years
Trial Details
ClinicalTrials.Gov Id: NCT06366464
Phase: 3
Lead Sponsor: Harmony Biosciences
Countries: United States, Canada, Europe, Australia
Additional Details: Please visit the TEMPO website for a current list of trial sites and for more information on this study.
Questions?
Contact: Ann Adee, Harmony Biosciences
Phone: 773-383-6258
Email: clinicaltrials@harmonybiosciences.com
