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
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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 - Closing soon
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 from across Australia and NZ)
Contact:
NOVA Trial — Clinical Trial Opportunity for the PWS Community | Australia
NSW - RPAH: clinicaltrials.boden@sydney.edu.au
Vic - Alfred: neurologyclinicaltrials@alfred.org.au
Qld - Princess Alexandra: diabendoresearch@health.qld.gov.auMore information: Flyer
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Sleep difficulties are common across the developmental disability community. Dr Baker and her team want to understand what matters most to you when it comes to sleep research.
Status: Recruiting
Type: Online survey
Who: People with PWS, and/or family/carers
Survey link: Participant Information Statement
Contact: ebaker@flinders.edu.au
More Information: FLYER
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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
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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 lifted, awaiting news of a new trial
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 Therapeutics Announces FDA Has Lifted the Clinical Hold on ARD-101 for the Treatment of Prader-Willi Syndrome and Will Host Webcast to Review the Available Exploratory Phase 3 HERO/OLE Data – Thu, 10/22/2026 - 16:05 -
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.auTitle: 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
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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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