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 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up to day 441

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

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