Status:

RECRUITING

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Lead Sponsor:

Harmony Biosciences Management, Inc.

Conditions:

Prader-Willi Syndrome

Eligibility:

All Genders

6+ years

Phase:

PHASE3

Brief Summary

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 ...

Detailed Description

The study will consist of an up to 45-day Screening/Baseline Period, a Double-Blind Treatment Period, and an optional Open-Label Extension Period. After completion of all Baseline assessments, patien...

Eligibility Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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

Key Trial Info

Start Date :

May 28 2024

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

July 1 2027

Estimated Enrollment :

134 Patients enrolled

Trial Details

Trial ID

NCT06366464

Start Date

May 28 2024

End Date

July 1 2027

Last Update

March 17 2026

Active Locations (54)

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Page 1 of 14 (54 locations)

1

Santa Monica Clinical Trials

Los Angeles, California, United States, 90025

2

Center of Excellence in Diabetes and Endocrinology

Sacramento, California, United States, 95821-2123

3

Rady Children's Hospital - Scan Diego

San Diego, California, United States, 92123

4

Colorado Children's Hospital

Aurora, Colorado, United States, 80045

A Study of Pitolisant in Patients With Prader-Willi Syndrome | DecenTrialz