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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Upcoming Trials

Past Trials

  • TRIAL TITLE: A Phase 2b, Double-blind, Randomized, Placebo-controlled, Multi-center, 16-week Dose Finding, Safety and Efficacy Study With Open-label Extension (OLE) Period of Tesomet in Subjects With Prader-Willi Syndrome

    This study will evaluate the safety and efficacy of Tesomet (tesofensine + metoprolol) in subjects with PWS. For the double-blind portion of the study, dosing will be initiated in a subgroup of adults who are 18-65 years of age. Following independent Data Monitoring Board review of subgroup safety data, and review and confirmation to proceed by FDA, enrollment of subjects <18 years of age will commence.

    SPONSOR: Saniona

    INDICATION: Prader-Willi Syndrome

    STUDY PROTOCOL: TM006

    PHASE: 2b

    STATUS: Completed

    RECRUITING PATIENTS: No

  • TRIAL TITLE: An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Prader-Willi Syndrome

    DESCRIPTION: NEU-2591-PWS-001 is an open-label study of the safety, tolerability, and pharmacokinetics (PK) of NNZ-2591 Oral Solution, 50 mg/mL in male and female children and adolescents with PWS ages 4 to 12 years. Participants receive treatment of orally administered NNZ-2591 for a total of 13 weeks.

    SPONSOR: Neuren Pharmaceuticals Ltd.

    INDICATION: Prader-Willi Syndrome

    STUDY PROTOCOL: NEU-2591-PWS-001

    PHASE: 2

    STATUS: Inactive

    RECRUITING PATIENTS: No