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 groupsLocations
54Australia
Queensland Children's Hospital BrisbaneQueensland, Australia
Royal Prince Alfred Hospital Camperdown Australia
Sydney Children's Hospital Randwick Australia
Children's Hospital at Westmead Westmead Australia
Sponsors and collaborators
Harmony Biosciences Management, Inc.
Lead sponsor
Source ClinicalTrials.gov