A Study of Pitolisant in Patients With Prader-Willi Syndrome
ConditionPrader-Willi Syndrome
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age6+
SponsorHarmony Biosciences Management, Inc.
About this trial
This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome.
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 objectives include assessing the impact of pitolisant on:
Irritable and disruptive behaviors Hyperphagia Other behavioral problems including social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech
Eligibility criteria
Qualifiers
Genetically confirmed diagnosis of PWS
Excessive daytime sleepiness
Has a consistent parent/caregiver (preferably the same person throughout the study) who is willing and able to complete the required study assessments.
In the opinion of the Investigator, the patient/parent(s)/caregiver(s)/legal guardian(s) are capable of understanding and complying with the requirements of the protocol and administration of oral study drug.
Disqualifiers
Has a diagnosis of sleep apnea (OSA, CSA) that is not adequately controlled
Has a diagnosis of hypersomnia due to another sleep/medical disorder
Participation in an interventional research study involving another investigational medication, device, or behavioral treatment within 30 days or 5 half-lives (whichever is longer) of the investigational medication prior to Screening
Trial design
Treatments tested in this trial
- Pitolisant tablet
- Placebo tablet
Treatment groups
134 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov