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
This trial does not accept healthy volunteersQualifiers
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
Design model
Parallel
Treatments tested in this trial
Pitolisant tablet
DrugPitolisant tablet
Placebo tablet
Other interventionPlacebo tablet
Treatment groups
134 Participants
are divided into 3 treatment groupsGroup A: Double-Blind Treatment Period PitolisantExperimental treatment 1 intervention
Group B: Double-Blind Treatment Period PlaceboPlacebo comparator 1 intervention
Group C: Open-Label Extension Period PitolisantOther 1 intervention
Trial outcomes
Primary outcomes
1
Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score
The PROMIS-SRI item bank consists of 13 items with a 5-point rating scale.
Time frame
Baseline and end of the Double Blind Treatment Period (Day 77)Secondary outcomes
1
Change in severity of irritable and disruptive behaviors as measured by the Aberrant Behavior Checklist-Community, Second Edition (ABC-C) Irritability domain
The ABC-C is a 58-item questionnaire, divided into 5 subscales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/Noncompliance, and Inappropriate Speech).
Time frame
Baseline and end of the Double Blind Treatment Period (Day 77)2
Change in overall severity of EDS as measured by the Caregiver Global Impression of Severity for Excessive Daytime Sleepiness (CaGI-S for EDS)
The CaGI-S for EDS is a 1-item, 5-point rating scale.
Time frame
Baseline and end of the Double Blind Treatment Period (Day 77)3
Change in overall severity of EDS as measured by the Clinical Global Impression of Severity for Excessive Daytime Sleepiness (CGI-S for EDS)
The CGI-S for EDS is a 1-item, 5-point rating scale.
Time frame
Baseline and end of the Double Blind Treatment Period (Day 77)4
Change in overall severity of irritable and disruptive behaviors as measured by the Caregiver Global Impression of Severity (CaGI-S) for Irritable and/or Disruptive Behaviors
The CaGI-S for Irritable and/or Disruptive Behaviors is a 1-item, 5-point rating scale.
Time frame
Baseline and end of the Double Blind Treatment Period (Day 77)Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov