Prader–Willi Syndrome (PWS)

Condition Description

A genetic disorder that causes obesity, intellectual disability, and shortness in height.

Prader-Willi syndrome is a genetic disorder usually caused by deletion of a part of chromosome 15 passed down by the father.

The most common symptoms of Prader-Willi syndrome are behavior problems, intellectual disability, and short stature. Hormonal symptoms include delayed puberty and constant hunger leading to obesity.

Active Trials

  • TRIAL TITLE: 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.

    DESCRIPTION: 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.

    SPONSOR: Harmony Biosciences, LLC

    INDICATION: Prader-Willi Syndrome

    STUDY PROTOCOL: HBS-101-CL-312

    PHASE: 3

    STATUS: Active

    RECRUITING PATIENTS: Yes

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