Clinical Trials Recruiting in Australia

Clinical trials tell us if a new therapy is safe and if it will help make life with PWS better.

The faster a clinical trial is completed, the sooner we know if the therapy works.   You can help by taking part in clinical trials and registries.  Your doctor and the study coordinator can help you decide if a trial is a good fit for you and your family.

Clinical trial resources:

Disclosure: PWFRA does not endorse or recommend any specific study, we aim to provide information about trials that people with PWS living in Australia can participate in.

PWS Clinical Trials

Currently recruiting

  • Status: Recruiting
    Full title: Gastrointestinal transit and safety of tirzepatide in people with PWS
    Type: Intervention (drug)
    Age: Adults
    Location: Sydney (RPAH)
    Contact: clinicaltrials.boden@sydney.edu.au
    More information: Recruitment video

  • Status: Recruiting
    Full title: TEMPO study; evaluating pitolisant for excessive daytime sleepiness in PWS (Harmony Biosciences)
    Type: Intervention (drug)
    Age: 6+
    Location: Brisbane, Sydney (travel available)
    Contact:
    Leapcure: lpcur.com/tempo-au
    Queensland Children’s Hospital: chq_pwsresearch@health.qld.gov.au
    Sydney Children’s Hospital Randwick: schn-schclinicaltrials@health.nsw.gov.au
    Sydney Children’s Hospital Westmead: schn-clinicaltrials@health.nsw.gov.au
    RPAH: clinicaltrials.boden@sydney.edu.au
    More information: Flyer

  • Status: Recruiting
    Full title: A Randomized, Double-Blind, Placebo-controlled Phase 2 Study to Assess Efficacy, Safety and Tolerability of BMB-101 Oral Solution for the Treatment of Patients with PWS
    Type: Intervention (drug)
    Age: 18 to 65 years
    Location: Melbourne, Sydney (travel available)
    Contact:
    Leapcure: lpcur.com/LC-PWS
    RPAH: clinicaltrials.boden@sydney.edu.au
    Alfred: neurologyclinicaltrials@alfred.org.au
    More information: Flyer

  • Status: Recruiting
    Full title: Understanding Family Perspectives on Clinical Trials for Prader-Willi Syndrome
    Type: Survey
    Age: All
    Location: National (online)
    Contact: diane.webster@praderwilli.org.au
    Surveys:
    Survey for families and carers
    Survey for people with PWS

  • Status: Recruiting
    Type: Survey
    Age: 12 to 65 years at time of anaesthetic
    Location: National (online)
    Contact: Complete the survey
    More information: Flyer

  • Status: Recruiting
    Full title: GeneEQUAL: Co-production with people with intellectual disability to achieve more inclusive, person-centered, and respectful healthcare
    Type: Observation (interviews)
    Age: Parents and carers, and/or young people with PWS (up to 28 years)
    Location: Discuss with study lead
    Contact: geneequal.com

 

Closed or paused

  • Status: Closed, awaiting results
    Type: Intervention (drug)
    Age: 18 to 30
    Location: Sydney (RPAH)
    Contact: clinicaltrials.boden@sydney.edu.au or 02 8627 6048
    More information: PWS flyer | Recruitment video

  • Status: FDA clinical hold
    Full title: The Hunger Elimination or Reduction Objective Trial (ARD101/Aardvark Therapeutics)
    Type: Intervention (drug)
    Age: 10 years and older
    Location: Sydney, Brisbane (travel available)
    More information: Aardvark press release | FAQ for parents and caregivers

  • Status: Closed, awaiting results
    Type: Observation and pedometer
    Age: 2 to 17
    Location: Melbourne (RCH)
    Contact: Anela Halilagic, 0478 059 555 or A.Halilagic@latrobe.edu.au

    Title: In their own voice: My Hunger Questionnaire (MY-HQ)
    Description:
    Status: Closed, awaiting results
    Type: Survey and interview
    Age: 10+
    Location: Online and Zoom
    Contact: elizabeth.roof@vanderbilt.edu
    More information: Project info | Flyer

 

PWS registries and biobanks

  • Status: Recruiting
    Type: Intervention (biological sample collection, surveys and assessments)
    Age: Any
    Location: National
    Contact: diagnosis.development@mcri.edu.au
    More information: Biobank flyer | Biobank EOI

  • Status: Recruiting
    Type: Observational (survey)
    Age: All ages
    Location: Online
    Contact: pwsregistry.org

  • Status: Recruiting
    Type: Observational (survey)
    Age: 0 to 25 years
    Location: Online
    Contact: jenny.downs@telethonkids.org.au

Please forward details of additional PWS studies recruiting in Australia to info@praderwilli.org.au

Last updated: October 2026

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