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Histamine H3 Receptor Antagonist

Pitolisant for Prader-Willi Syndrome

Phase 3
Recruiting
Research Sponsored by Harmony Biosciences, LLC
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to day 441
Awards & highlights
Pivotal Trial

Summary

This trial is testing a medication called pitolisant to see if it can help patients with Prader-Willi syndrome who experience excessive daytime sleepiness. The study will also look at how pitolis

Who is the study for?
This trial is for patients aged 6 and older with Prader-Willi Syndrome, specifically to address excessive daytime sleepiness. Details about who can join or reasons for exclusion are not provided.
What is being tested?
The study tests the effectiveness of a drug called Pitolisant compared to a placebo in reducing excessive daytime sleepiness in Prader-Willi Syndrome patients. It's randomized and double-blind, meaning neither participants nor researchers know who gets the real drug versus the placebo.
What are the potential side effects?
Specific side effects of Pitolisant are not listed here, but common ones may include headache, insomnia, nausea, and irritability based on its use in treating other conditions.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to day 441
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to day 441 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change in severity of EDS as measured by Patient-Reported Outcomes Measurement Information System Bank v1.0 - Sleep-Related Impairment (PROMIS-SRI) T-score
Secondary study objectives
Change in overall severity of EDS as measured by the Caregiver Global Impression of Severity for Excessive Daytime Sleepiness (CaGI-S for EDS)
Change in overall severity of EDS as measured by the Clinical Global Impression of Severity for Excessive Daytime Sleepiness (CGI-S for EDS)
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
+5 more

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: Open-Label Extension Period PitolisantExperimental Treatment1 Intervention
Pitolisant tablets administered once daily in the morning upon wakening
Group II: Double-Blind Treatment Period PitolisantExperimental Treatment1 Intervention
Pitolisant tablets administered once daily in the morning upon wakening
Group III: Double-Blind Treatment Period PlaceboPlacebo Group1 Intervention
Matching placebo administered tablets once daily in the morning upon wakening

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Who is running the clinical trial?

Harmony Biosciences, LLCLead Sponsor
8 Previous Clinical Trials
1,884 Total Patients Enrolled
1 Trials studying Prader-Willi Syndrome
65 Patients Enrolled for Prader-Willi Syndrome
~89 spots leftby Jul 2026
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