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: 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: Active

    Enrolling: Yes

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Upcoming Trials

Past Trials

  • TRIAL TITLE: Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome (PWS)

    DESCRIPTION: This study aimed to evaluate the safety and efficacy of Tesomet (tesofensine + metoprolol) in subjects with PWS.

    SPONSOR: Saniona

    INDICATION: Prader-Willi Syndrome

    STUDY PROTOCOL: TM006

    PHASE: 2b

    STATUS: Completed