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
| Study | Type | Age | Location | Contact/Study Link | Status |
|---|---|---|---|---|---|
| Acamprosate; investigating a new target for treatment in PWS PWS FLYER Recruitment Video |
Intervention - Drug | 18-30 | Sydney RPAH | clinicaltrials.boden@sydney.edu.au or 02 8627 6048 | Recruiting |
| Tania Markovic; Gastrointestinal transit and safety of Tirzepatide in people with PWS Recruitment Video |
Intervention - Drug | adults | Sydney RPAH | clinicaltrials.boden@sydney.edu.au | Recruiting |
| GeneEQUAL: Co-production with people with intellectual disability to achieve more inclusive, person-centered, and respectful healthcare Study Link |
Observation (interviews) | parents/carers and/or young people with PWS (up to 28yo) | discuss with study lead | Contact: https://geneequal.com/ | Recruiting |
| Harmony Biosciences; TEMPO study; evaluating pitolisant for excessive daytime sleepiness in PWS |
Intervention (drug) | TBC | Qld, Sydney |
Queensland Children’s Hospital
Megan Angliss. Email: chq_pwsresearch@health.qld.gov.au
Sydney Children’s Hospital Randwick Email: schn-schclinicaltrials@health.nsw.gov.au Sydney Children’s Hospital Westmead. Investigator: Dr. Yoon Hi Cho, Email: schn-clinicaltrials@health.nsw.gov.au RPAH: Study Coordinator Tegan Picone email: tegan.picone@sydney.edu.au |
Recruiting |
| ARD-101 for Treatment of PWS: The Hunger Elimination or Reduction Objective Trial (HERO) | Intervention (drug) | 13 Years and older | NSW (Sydney) and QLD (Brisbane) |
Queensland Children’s Hospital:
Ellen Taylor, Email: CHQ_CCTRND@health.qld.gov.au or ph: 07 3069 7532
Sydney Children’s Hospital Network Westmead: TBA Royal Prince Alfred Hospital: Tegan.Picone@sydney.edu.au |
Recruiting |
| Growth and Energy requirements in PWS | Observation & Pedometer | Child with PWS, 2-17 years old | Royal Children's Hospital, Melbourne | Anela Halilagic, 0478 059 555 or email: A.Halilagic@latrobe.edu.au | Recruiting |
| In their Own Voice: My Hunger Questionnaire (MY-HQ) MY-HQ Project Info MyHQ Flyer |
Survey & interview | 10+ | Online & Zoom | email: elizabeth.roof@vanderbilt.edu | Recruiting |
| 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 Prader-Willi Syndrome (PWS) | Intervention (drug) | 18-65 years | Melbourne (Vic) and Sydney (NSW), interstate travel may be supported. | TBA | Coming Soon |
| PWRFA: Understanding Family Perspectives on Clinical Trials for Prader- Willi Syndrome | Survey | All | National (online) | Email: diane.webster@praderwilli.org.auor go directly to the survey: https://www.surveymonkey.com/r/JLCHTBG | Recruiting |
| IPWSO: Response to anaesthetic in people with PWS Download Flyer |
Survey | 12 - 65 y at time of anaesthetic | National Online | https://redcap.slhd.nsw.gov.au/surveys/?s=JR7YAELX9M77TJMK | Recruiting |
PWS Registries and Biobanks
| Study | Type | Age | Location | Contact/Study Link | Status |
|---|---|---|---|---|---|
| Australian Chromosome 15 Biobank Biobank Flyer Biobank EIO |
Intervention - Biological sample collection, surveys and assessments | Any | National | diagnosis.development@mcri.edu.au | Recruiting |
| Global PWS Registry |
Observational - survey | All ages | Online | www.pwsregistry.org | Recruiting |
| Australasian Prader Willi Database | Observational - survey | 0-25 years | Online | Contact: jenny.downs@telethonkids.org.au | Recruiting |
Please forward details of additional PWS studies recruiting in Australia to info@praderwilli.org.au
Last updated: 04 November 2025
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