Testing Pitolisant against a placebo for prader-willi syndrome
Official title: A Study of Pitolisant in Patients With Prader-Willi Syndrome
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
- Phase 3
- 3 groups
- Sites in Thornhill and Toronto
- Recruiting
Interventions
- Medication
Pitolisant tablet
Pitolisant tablet
- Other intervention
Placebo tablet
Placebo tablet
Canadian Sites (2)
1 of 2 recruiting
- Recruiting
AMNDX Inc.
Thornhill, Ontario
- Withdrawn
Jodha Tishon Inc.
Toronto, Ontario
Eligibility Criteria
See who this study is looking for7 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +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 InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →, the patient/parent(s)/caregiver(s)/legal guardian(s) are capable of understanding and complying with the requirements of the protocolProtocolThe detailed plan a study must follow.Read more → and administration of oral study drug.
Exclusion
- −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 interventionalInterventional studyA study where participants are given something to see what happens.Read more → 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 ScreeningScreeningThe checks done before joining, to see whether a study fits.Read more →
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 3Phase 3A large study comparing a treatment against the current standard.Read more → — a large study comparing this against the current standard, usually across many hospitals.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 3 groups in this study.
Groups A and C receive Pitolisant tablet.
Registry label: A: Double-Blind Treatment Period Pitolisant · C: Open-Label Extension Period Pitolisant
Group B, the placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group, receives Placebo tablet.
Registry label: B: Double-Blind Treatment Period Placebo
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
One group receives a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → — a dummy treatment with no active medicine in it.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists a minimum age of 6 years, with no upper limit given.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 134 people.
The study is currently expected to finish around April 2028.
The main measurement is taken over: BaselineBaselineYour starting measurements, taken before treatment begins.Read more → and end of the Double BlindDouble-blindNeither you nor the study team knows which group you are in.Read more → Treatment Period (Day 77).
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score — measured over BaselineBaselineYour starting measurements, taken before treatment begins.Read more → and end of the Double BlindDouble-blindNeither you nor the study team knows which group you are in.Read more → Treatment Period (Day 77).
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT06366464 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About Pitolisant tabletDrug
Pitolisant tablet
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About Placebo tabletOther
Placebo tablet
From the trial registry — its own words, unedited.
What a other is here: An intervention the registry did not place in another category.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: a minimum age of 6 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
Yes. This study includes a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group — a dummy treatment with no active medicine in it.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists a minimum age of 6 years, with no upper limit given.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: BaselineBaselineYour starting measurements, taken before treatment begins.Read more → and end of the Double BlindDouble-blindNeither you nor the study team knows which group you are in.Read more → Treatment Period (Day 77).
The study as a whole is currently expected to finish around 2028-04.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 134 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 5 locations, including: Thornhill, Ontario, Canada; Toronto, Ontario, Canada; Rochester, Minnesota, United States; Brooklyn, New York, United States; The Bronx, New York, United States.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
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
Other Sites (3)
Mayo Clinic-PPDS
Rochester, Minnesota, United States
Maimonides Medical Center
Brooklyn, New York, United States
Montefiore Medical Center
The Bronx, New York, United States
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.